Sun 12 Nov
7:00 - 18:00
Registration Hours
8:00 - 12:00
Short Course Morning Sessions
Health Economic Modeling in R: A Hands-on Introduction
Level: Introductory
Track: Economic Evaluation
Separate registration required. This highly practical course will outline the computational and transparency advantages of using R, for those used to health economic modelling using Microsoft Excel. This course explores the use of R for health economic modelling in the context of health economics and outcomes research (HEOR) and faculty will guide the participants through practical examples of HEOR. The faculty are expert speakers who have diverse experience in academia, national Health Technology Assessment agencies (NICE, NCPE), and industry. The faculty will lead participants through practical examples of health economic modelling including using R for Markov models from deterministic analysis through to probabilistic sensitivity analysis and EVPI. Additional useful packages for modelling using R will also be discussed. All sessions will interchange between descriptive lectures and hands-on exercises. Participants will be provided with materials, including model examples in R and information on where to go for further learning.
This course is designed for those with some familiarity with modelling techniques, such as the concepts of discrete time cohort Markov models and probabilistic sensitivity analysis, but familiarity with R coding is not required.
Attendees will require a laptop with RStudio (v1.1.0 or higher) and R (v4.2.1 or higher) downloaded and installed.
Faculty Member
Gianluca Baio, PhD
University College London, London, United Kingdom
Gianluca Baio is a professor of Statistics and Health Economics in the Department of Statistical Science at University College London (UK). Gianluca graduated in Statistics and Economics from the University of Florence (Italy). He then completed a PhD programme in Applied Statistics again at the University of Florence, after a period at the Program on the Pharmaceutical Industry at the MIT Sloan School of Management, Cambridge (USA); he then worked as a Research Fellow and then Temporary Lecturer in the Department of Statistical Science at University College London (UK). Gianluca's main interests are in Bayesian statistical modelling for cost effectiveness analysis and decision-making problems in the health systems, hierarchical/multilevel models and causal inference using the decision-theoretic approach. Gianluca leads the Statistics for Health Economic Evaluation research group within the department of Statistical Science and he is the co-director of UCL MSc Programme in Health Economics and Decision Science.
Rose Hart, PhD
Lumanity Inc., Sheffield, United Kingdom
Rose Hart is a senior health economist at Lumanity, where she has been part of the modeling team for over 5 years with a focus towards advanced analytics and strategic solutions. She is experienced in developing health economic models for early-phase cost effectiveness and for health technology assessment. As a specialist in R modelling, with and without Shiny interface functionality, she is responsible for internal Shiny training and is a development leave in expanding Lumanity’s intRface™ services. Rose previously worked in the BioPharm team at GlaxoSmithKline and researched for her PhD at the University of Sheffield.
Felicity Lamrock, PhD
Queens University Belfast, Belfast, ANT, United Kingdom
Dr. Felicity Lamrock is a Lecturer in Data Analytics at Queen’s University Belfast. She is currently the Director of the Northern Ireland Centre for Health Analytics and Decision Science (NI-CHADS) with a focus on the analysis of health data for decision modelling. Current projects include a range of disease areas including cancer, rare diseases, diabetes, COVID-19, and cardiovascular disease. Felicity was previously a statistician at the National Centre for Pharmacoeconomics (NCPE) working with a team of pharmacists and clinicians on Health Technology Assessments to advise the Health Service Executive on the recommendation of new drug therapies in Ireland. She remains involved with NCPE as a statistical advisor and is exploring how Northern Ireland could benefit from more decision modelling/pharmacoecononomic assessment.
Howard Thom, MSc, PhD
University of Bristol, Bristol, UK; Clifton Insight, Bristol, SOM, United Kingdom
Howard Thom is a Senior Lecturer in Health Economics at the University of Bristol and Senior Director at the consultancy Clifton Insight. His research interests are value of information, uncertainty in economic models, network meta-analysis, and R for health economics.
Developing Decision-Grade Real-World Evidence
Level: Intermediate
Track: Real World Data & Information Systems
Separate registration required. In this course, participants will be guided through a hands-on analysis of real-world data to develop decision-grade real-world evidence (RWE) that could be used to support an indication expansion. The first section of the course focuses on what makes RWE “decision-grade.” We will review the most recent RWE frameworks and guidelines set by regulatory agencies and professional organizations, and we will examine case studies in which these guidelines were used in regulatory and HTA approval. The second half of the course is an active workshop where participants will use principles from the first half of the course to execute a decision-grade RWE study. Participants will be guided step-by-step in using a software platform that will enable them to work within a longitudinal US insurance claims database with anonymized patients. After the study has been executed, we will discuss how these results could be communicated to decision makers. Participants should come with a laptop with Google ChromeTM installed.
PREREQUISITE : Students are expected to be familiar with relevant concepts and methodologies for analyzing real-world data, but this course does not require specific programming skills.
Faculty Member
Dorothee Bartels, PhD, MSc
Aetion, Inc., Neuss, Germany
Dorothee Bartels is a trained epidemiologist and is currently the chief digital officer at Aetion. She has more than 15 years of experience in pharma industry as
head of Real-World Evidence (RWE) and Digital Sciences at UCB Biosciences GmbH, as corporate head of Global Epidemiology at Boehringer Ingelheim, and as Chief digital science officer in the innovation Lab, Bi X. She also was the Clinical and Real-World Data Strategy lead at X, Moonshot (Alphabet Inc., former Google X).
She maintains appointments as associate professor for Public Health and Epidemiology at Hannover Medical School (MHH), as adjunct professor for Epidemiology and Biostatistics at McGill University and teaches in the course “Successful Implementation of Digital Health” at the Institute for Communication and Leadership (Switzerland). She also holds several roles on advisory boards.
Her aim is to enhance evidence generation based on real-world data, including patient-generated health data (digital epidemiology), and applying ML/AI in combination with established epidemiological statistical methods.
Shirley Wang, PhD, MSc
Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA
Dr. Wang is an associate professor at Brigham and Women’s Hospital, Harvard Medical School and lead epidemiologist for the Food and Drug Administration’s (FDA) Sentinel Innovation Center. She leads the Meta-Research in Pharmacoepidemiology program, with recent projects aimed at improving the transparency, reproducibility and robustness of evidence from healthcare databases (www.repeatinitiative.org) and informing when and how real-world evidence studies can draw causal conclusions to inform regulatory or other healthcare decision-making (www.rctduplicate.org). She is currently PI on multiple NIH R01s and is also funded by FDA. Her methods work has received 3 awards from international societies.
Risk-Sharing/Performance-Based Arrangements in Developing Countries
Level: Intermediate
Track: Health Policy & Regulatory
Separate registration required. During recent years, Managed Entry Agreements (MEAs) have become instrumental in ensuring the access of innovative medicines. This course is designed for healthcare professionals (including public decision-makers, academia, and industry) involved in pricing and reimbursement decisions who are wishing to understand the applicability and technical aspects of managed entry agreements (MEAs) in countries with severe economic constraints and explicit cost-effectiveness criterion. The topic will be introduced with key features of pricing and reimbursement systems in representative countries to understand why special methods are needed to facilitate evidence-based reimbursement policies of new health technologies. Faculty will present an economic model to explain the methodology and implications of managed entry agreements in cost-effectiveness and budget impact analysis. Participants will then have the opportunity to apply what they have learned through a hands-on exercise on making pricing and reimbursement decisions. A decision algorithm will be presented to support evidence and value-based policy decisions of high-cost new technologies. A series of password protected economic models will add more and more complexity to a pragmatic case study on a new pharmaceutical product in oncology. To close the course faculty will lead a discussion on the applicability of a pragmatic decision tool illustrating the pros and cons of different managed entry agreements and their usefulness in CEE settings. Participants who wish to gain hands-on experience must bring their laptops with Microsoft Excel for Windows installed.
Faculty Member
Rok Hren, PhD, MSc IHP (HE)
University of Ljubljana, Ljubljana, Slovenia
Rok Hren has more than 15 years of commercial experience in pharmaceutical industry, with more than 12 years on a board level in both (i) line management, which has included full P&L responsibility for operations in Slovenia and Romania, and (ii) leadership regional functions in Central and Eastern Europe. He regularly presents on the topic of pharmaceutical economics and policies at conferences in Europe and is well experienced in healthcare media business.
He received his PhD in Physiology and Biophysics from Dalhousie University, Canada and MSc in International Health Policy (Health Economics) with Distinction from London School of Economics and Political Science, UK while he was a post-Doctoral Fellow at Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah Medical School, USA. He is also a professor at the University of Ljubljana and past president of ISPOR Slovenia Regional Chapter. In total, his publications gathered 300/394 citations (excluding self-citations) in WoS/Scopus (as of September 10, 2016).
Katarzyna Kolasa, PhD
Kozminski University, Warsaw, MZ, Poland
Katarzyna Kolasa PhD
Professor of Health Economics at Kozminski University Warsaw Poland
Digital Health Start Me Up Course leader
ISPOR Digital Health SIG founder
“The Digital Transformation of the Healthcare System” Routledge; 1st edition (July 31, 2023)
Driven with passion to improve healthcare, Katarzyna has focused her academic and busi-ness career on the health economics.
She has been working with multiple pricing & reimbursement challenges worldwide for the last 25 years, while holding various Regional and Global leadership positions at Astra Zeneca, BMS, Biogen Idec, Lundbeck, GE Healthcare, Straub Medical, BD and the Swedish county council of Kalmar. Katarzyna is mentor and consultant to start ups involved in the development of innovative medical devices and digital health solutions from both Holland and Poland.
Since 2000 she has been academic teacher and supervisor for over 30 MBA and Phd stu-dents. In partnership with the Polish Medical Research Agency, Deloitte Digital and the Polish Central Hospital of Ministry of Interior Affairs she founded the first Digital Health six months educational program designed for digital transformation leaders working in the healthcare sector. Katarzyna developed innovative Master Program Health Economics & Big Data (HEBDA) which first edition was financed by EU Power Grant 2018 as well.
She is the founder of the Global Special Interest Group Digital Health and short courses “The Role of Digital Endpoints in the Value Generation for Health Technologies”, “Risk-Sharing/Performance-Based Arrangements in Developing Countries” at International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She is the current member of Edu-cational and post member of Health Science Policy Councils at ISPOR as well.
Katarzyna has dedicated her academic research towards the methodological advancements into the value assessment of pharmaceuticals, medical devices, and digital health solutions. Passionate about Big Data, she led the first project of machine learning adaptation for the optimal utilization of CT scanners granted by the Polish Ministry of Health as well. Since January 2022, she is the leader of AI special interest group at the Polish Chamber of Physi-cians. With the patronage of the Polish Parliamentary Commission for Innovation & Digitali-zation, she organized the first dialog about the societal preferences towards the adoption of AI in the healthcare in Poland.
Being coauthor of more than 50 IF publications, she presented her research at more than 60 international scientific conferences. As of 2022, Google Scholar reports over 730 cita-tions to her work.
Bertalan Nemeth, PhD
Syreon Research Institute, Budapest, Hungary
Bertalan Németh PhD graduated from the Corvinus University of Budapest (MSc in Quantitative economics and Operation research), the Eötvös Loránd University (Pharmaceutical economics and drug policies), and the Semmelweis University School of PhD Studies. Between 2010 and 2015 he was a health economist at the Hungarian HTA office. Since August 2015 Bertalan has been a senior health economist, and since 2019 a Principal Researcher at Syreon Research Institute. Bertalan is responsible for strategic consulting, and he is involved in various projects that include modelling, economic evaluation in health, health technology assessment and health statistics as well. Bertalan was the president of the ISPOR Hungary Chapter and was the chair of the ISPOR CEE Consortium. He was a participant in the international EUnetHTA project, the ISPOR HTA Roundtable Europe, and the Scientific Committee of the META Conference. Bertalan was also a faculty member of the global ISPOR HTA Training and was the module leader of Health Technology Assessment for the MSc program at Eötvös Loránd University.
Network Meta-Analysis in Relative Effectiveness Research
Level: Intermediate
Track: Study Approaches
Separate registration required. For several medical questions of interest, many treatment options exist for the same indication. These treatments may have been compared against placebo or against each other in clinical trials. Knowing whether one specific treatment is better than placebo or some other specific comparator is only a fragment of the big picture, which should incorporate all available information. Ideally, one would know how all the treatment options rank against each other and the level of differences in treatment effects between all the available options. Network meta-analysis provides an integrated and unified method that incorporates all direct and indirect comparative evidence about treatments. Based in part on the ISPOR Task Force Reports on Indirect Treatment Comparisons, the fundamentals and concepts of network meta-analysis will be presented. The evaluation of networks presents special challenges and caveats, which will also be highlighted in this course. The material is motivated by instructive and concrete examples. The ISPOR-AMCP-NPC questionnaire for assessing the credibility of a network meta-analysis will also be introduced.
This course requires at least a basic knowledge of meta-analysis and statistics.
Faculty Member
Sarah Goring, MSc
SMG Outcomes Research, Vancouver, BC, Canada
Sarah Goring has nearly 20 years of experience in health economics and outcomes research. Sarah is currently an independent consultant, providing scientific leadership on a broad range of studies including epidemiological and burden of illness studies, health services research, systematic reviews, and evidence synthesis. Sarah has a B.Sc. in mathematics from the University of Victoria and an M.Sc. in health care and epidemiology from the University of British Columbia. Sarah is a co-editor of a Springer textbook on Health Services Research, and has co-authored over 70 research contributions.
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Introduction to Health Technology Assessment
Level: Introductory
Track: Health Technology Assessment
Separate registration required. This introductory course is designed to teach academic researchers, health policy decision makers, manufacturers, and clinicians about the key elements, methods, and language of health technology assessment (HTA). The course provides an overview of basic HTA principles including benefit assessment (biostatistics, clinical epidemiology, patient-relevant outcomes, risk-benefit assessment), economic evaluation (costing, cost-effectiveness analysis, pharmacoeconomic modeling, budget impact analysis, resource allocation), and ELSI (ethical, legal, and social implications). Using real world examples covering both drugs and devices, the course will review the practical steps involved in developing and using HTA reports in different countries and healthcare systems. Discussion with participants will focus on the implementation of HTA in health care decision making and stakeholder perspectives.
This course is suitable for those with little or no experience with HTA.
Faculty Member
Petra Schnell-Inderst, MPH, PhD, Dipl. Biol
UMIT TIROL - University for Health Sciences and Technology, Hall i. T., 7, Austria
Dr. Dipl.-Biol. Schnell-Inderst, MPH, is a Senior Scientist at the Institute of Public Health, Medical Decision Making and Health Technology Assessment at UMIT TIROL - University for Health Sciences and Technology in Hall i. T., Austria. She heads the Program on Health Technology Assessment at the Institute.
Her main research interests are in health technology assessment methodology with a focus on medical devices (see www.medtechta.eu. www.core-de.eu), digital health technologies, and public health interventions (screening programs).
Uwe Siebert, MD, MPH, MSc, ScD
UMIT TIROL - University for Health Sciences and Technology, Hall in Tirol, Austria. ONCOTYROL - Center for Personalized Cancer Medicine, Innsbruck, Austria. Harvard T.H. Chan School of Public Health and Harvard Medical School, Hall in Tirol, 7, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a Professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), Chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL - University for Health Sciences and Technology in Austria and Director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also Adjunct Professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in Decision Sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE, EUCAPA, PREMIO COLLAB). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (>30,000 citations, H index >80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
Introduction to Machine Learning Methods
Level: Intermediate
Track: Methodological & Statistical Research
Healthcare data are often available to payers and healthcare systems in real time, but are massive, high dimensional, and complex. Artificial intelligence and machine learning merge statistics, computer science, and information theory and offer powerful computational tools to enhance the extraction of useful information from complex healthcare data and prediction accuracy. This course gives an overview of basic machine learning concepts and introduces a few commonly used machine learning techniques and their practical applications in healthcare and pharmaceutical outcomes research. Participants will be introduced to foundational principles and concepts of statistical machine learning, then be provided with several specific machine learning techniques and their applications in health and pharmaceutical outcomes research. The course faculty will use R or Radiant to demonstrate several machine learning methods such as penalized regression and tree-based methods, as well as techniques for dimension reduction/feature selection. Participants will have hands-on practical experiences with machine learning and gain experience interpreting and evaluating the results and prediction performance that comes from machine learning modeling. Distinguishing prediction modeling from causal inference research in pharmacoepidemiology will be also presented and discussed. This is an entry-level course but is designed for those with some familiarity with traditional statistical modeling techniques (eg, linear regression, logistic regression).
PREREQUISITES: To get the most out of the course, students should have a basic statistical background. Participants who wish to gain hands-on experience are required to bring their laptops with Radiant (https://radiant-rstats.github.io/docs/install.html) installed.
Faculty Member
Wei-Hsuan Lo-Ciganic, PhD, MSPharm, MS
University of Florida, Gainesville, FL, USA
Dr. Wei-Hsuan Jenny Lo-Ciganic is a pharmacoepidemiologist and associate professor in the Department of Pharmaceutical Outcomes and Policy at the University of Florida College of Pharmacy. Her research agenda focuses on drug safety and addiction. Dr. Lo-Ciganic has extensive experience applying advanced predictive analytics including machine learning and trajectory modeling with large healthcare datasets. She conducts research to develop risk prediction algorithms and tools, and practical intervention applications for use in real-world settings to improve health outcomes and patient care. She is also a core faculty member in the Center for Drug Evaluation and Safety (CoDES) at the University of Florida College of Pharmacy.
William Padula, PhD, MSc, MS
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is Assistant Professor of Pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. He is a Co-Founder & Principal at Stage Analytics. His research focuses on the theoretical foundations of medical cost-effectiveness analysis and applications of machine learning. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, Co-Chair of the ISPOR Machine Learning Task Force, and is an Associate Editor for Value in Health.
Budget Impact Analysis I: A 6-Step Approach
Level: Introductory
Track: Economic Evaluation
Separate registration required. This course will describe the methods used to estimate the budget impact of a new health care technology and will present six basic steps for estimating budget impact: (1) estimating the target population; (2) selecting a time horizon; (3) identifying current and projected treatment mix; (4) estimating current and future drug costs; (5) estimating change in disease-related costs; and (6) estimating and presenting changes in annual budget impact and health outcomes. Both static and dynamic methods for estimating the budget and health impact of adding a new drug to a health plan formulary will be presented. These six steps will be illustrated using actual budget impact models.
This course is designed for those with some experience with pharmacoeconomic analysis.
Faculty Member
Thor-Henrik Brodtkorb, PhD
RTI Health Solutions, Ljungskile, O, Sweden
Thor-Henrik Brodtkorb, PhD, is executive director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 12 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision-analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and ISPOR.
At RTI-HS, Dr. Brodtkorb leads the development of cost-effectiveness, cost-utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Stephanie Earnshaw, PhD, MS
RTI Health Solutions, Pittsboro, NC, USA
Stephanie Earnshaw is senior vice president of Health Economics at RTI Health Solutions (RTI HS). She received her PhD in Industrial Engineering at North Carolina State University and has performed health outcomes and health services research for over 30 years, 24 of which have been with RTI-HS. She has presented workshops and various courses on decision-analytic modeling techniques for pharmaceutical companies and organizations such as ISPOR, the Academy of Managed Care Pharmacy (AMCP), and the Centers for Disease Control and Prevention (CDC). Dr. Earnshaw has served on the ISPOR Board of Directors and as Chair of the Audit Committee and is the current Educational Council Chair. She has held an Adjunct Faculty appointment at the University of North Carolina’s Eshelman School of Pharmacy, Division of Pharmaceutical Outcomes and Policy, is honored as a Distinguished Alumni in Industrial and Systems Engineering at North Carolina State University and is one of the lead authors of “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Dr. Earnshaw’s research focus is in applying decision-analysis techniques to industry-related issues and health care problems. Her areas of specialization include mathematical programming (constrained optimization), network optimization, and Markov, simulation, and other state transition modeling. In addition to developing budget-impact and cost-effectiveness models to support health technologies for the pharmaceutical, biotechnology, and diagnostic and medical device industry, she has developed innovative mathematical models using these methods to determine pricing strategy, predict clinical outcomes, allocate resources, and cost care pathways particularly in support of medical diagnostics. Therapeutic areas include cardiovascular disease, gastrointestinal disorders, respiratory disease, acute care, infectious disease, osteoporosis, vaccines, and oncology. She is a member of ISPOR and the Institute for Operations Research and the Management Sciences. She has presented her work at professional conferences and has published in several peer-reviewed journals.
C. Daniel Mullins, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor at the University of Maryland School of Pharmacy. He is founder and executive director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program. He received his BS in Economics from M.I.T. and his PhD in Economics from Duke University. His research and teaching focus on comparative effectiveness research (CER) and patient-centered outcomes research (PCOR). Dr. Mullins has received funding as a Principal Investigator from AHRQ, FDA, NHLBI, NIA, NIMHD, Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. He is the lead for the Community & Collaboration (C&C) Core of the University of Maryland Institute for Clinical and Translational Research (ICTR) and co-lead of the C&C Core for Johns Hopkins’ CTSA.
Professor Mullins is 1 of 2 editors-in-chief for Value in Health and is author of over 325 peer-reviewed articles and book chapters. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. He is a past recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award, and the ISPOR Marilyn Dix Smith Leadership Award.
Estimating Health-State Utility for Economic Models in Clinical Trials and Real-World Studies
Level: Introductory
Track: Patient-Centered Research
Separate registration required. Health state utility (HSU) estimates are among the most important and uncertain data inputs in cost-utility models which are increasingly being used to inform health technology assessment, pricing, and reimbursement decisions in many countries. This course will provide an in-depth consideration of best practice in the collection of health utility data in clinical trials, real-world and other studies, to provide high quality HSU estimates appropriate for economic modeling. Centered on the ISPOR Outcomes Research Guideline, Collecting Health-State Utility Estimates for Economic Models in Clinical Studies (Wolowacz et al., 2016), the course will address key challenges surrounding study design, data collection and analysis. This will include how to anticipate and address common issues that may affect data quality, alignment with the needs of economic model, acceptability to the model audience, and how to apply good research practices for HSU estimation in future research. The course will also address issues associated with collection of utility data for rare diseases and from special populations including cognitively impaired and pediatric populations. The course will be of value for researchers actively involved in the design or implementation of HSU data collection or analysis, those involved in patient-reported outcomes research, economic modeling, economic evaluation, or health technology assessment.
The course will not cover in any depth the fundamentals of utility theory, development of generic or condition-specific preference-based multi-attribute utility instruments, or how to perform time trade-off or standard gamble experiments. Nor will it cover statistical methods for mapping/cross-walking from a condition-specific HRQL measure. Although these topics will be touched on in overview, the focus of this course will be on optimizing the collection of utility data to provide HSU estimates for economic models.
Faculty Member
Lynda Doward, MRes
RTI Health Solutions, Manchester, United Kingdom
Ms. Doward has over 30 years of experience conducting patient-centered outcomes research
including the provision of strategic advice to pharmaceutical companies in the incorporation of
the patient voice into drug development programs. Ms. Doward is an expert in the development
of clinical outcome assessment (COA) strategies including the development of patient-centered
clinical trial endpoints, the implementation of patient-reported and other COA outcome measures
in clinical trial programs, and the inclusion of PRO and other COA value messages at key drug
development hurdles. Ms. Doward has extensive experience in supporting pharmaceutical clients
in their COA-related submissions to regulatory agencies in Europe and the US and advises on
health-utility measurement strategies for reimbursement agencies in Europe. Ms. Doward has led
the development of over 40 COA questionnaires that have been adapted and validated for use in
over 60 languages worldwide.
Ms. Doward currently serves on the ISPOR COA Special Interest Group (leadership committee)
and the ISPOR Patient Council (member) and was a member of the leadership committee of the
completed ISPOR Good Research Practices Task Force for the measurement of health state
utilities in clinical trials. Ms. Doward has acted as a consultant to the World Health Organization
and has served as a Research Advisor to the UK Department of Health, and medical charities in
the United Kingdom.
Emma Hawe, MSc
RTI Health Solutions, Manchester, United Kingdom
Emma Hawe, MSc, is Head of Data Analytics and Design Strategy at RTI-HS. She has over 20 years of experience as a statistician within consulting, regulatory, and academic environments. Ms. Hawe is experienced in the application of novel and standard statistical methodologies to large medical/biological data sets in diverse therapeutic areas. Her role at RTI-HS includes planning, executing, performing, and interpreting the analysis of a variety of studies, including systematic literature reviews, meta-analysis, epidemiology, health economics, and clinical trials. Prior to joining RTI-HS, Ms. Hawe was Head of Statistics at the Office of Health Economics, where she managed a variety of different projects, including burden-of-illness studies, multicriteria decision analysis, and policy-orientated projects for pharmaceutical companies, pharmaceutical trade associations, and overseas governments. Ms. Hawe has conducted many types of statistical analyses, including network meta analysis, survival analysis, factor analysis, analysis of utilities including mapping, multivariate modeling, and database analysis including the analysis of Hospital Episode Statistics (HES). She has successfully led and managed projects for a variety of different clients and contributed to many more. In addition, Ms. Hawe has experience with a variety of different statistical packages and programming languages, including R, STATA, SAS, and SQL. Ms. Hawe is author of the Office of Health Economics guide to UK health and health care statistics, a comprehensive guide to health statistics in the UK, and author of more than 75 publications in peer-reviewed journals. Previous positions have involved the statistical analysis of the combined effects of genetics and the environment on cardiovascular disease, and the study of births and infant mortality data in England and Wales over a 20-year period.
Andrew Lloyd, PhD
Acaster Lloyd Consulting Ltd, London, United Kingdom
Sorrel Wolowacz, PhD
RTI Health Solutions, Manchester, United Kingdom
Sorrel Wolowacz, PhD, is Head of European Health Economics at RTI-HS, with 22 years of experience in health economics research and consulting. Her research focuses primarily on economic modelling, health utility estimation, observational studies, and health technology appraisal submissions. Dr. Wolowacz is a member of the editorial board for the Journal of Comparative Effectiveness Research and was co-chair of the ISPOR Good Research Practices Task Force addressing Measurement of Health State Utility Values for Economic Models in Clinical Studies and is a member of the ISPOR Oncology Special Interest Group.
Statistical Methods for Health Economics & Outcomes Research
Level: Intermediate
Track: Economic Evaluation
Separate registration required. This course will provide an introduction to statistical concepts with an emphasis on the use of techniques commonly employed in health economics and outcomes research. Faculty will begin by defining statistics, then introducing the concept of random variables and probability before proceeding to discuss the foundations of statistical inference (estimation and the testing of hypotheses). This is followed by bootstrapping, statistics in cost-effectiveness analysis and generalized linear modeling (for cost and utility outcomes). The differences between a classical (frequentist) approach to statistics and a Bayesian view of probability will also be outlined.
This course is intended for participants with little (or rusty!) statistical training.
Faculty Member
Derrick Bennett, MSc, PhD, CStat
University of Oxford, Headington, Oxford, United Kingdom
Derrick Bennett is Associate Professor in Medical Statistics and Epidemiology. He has a BSc (Hons) in Mathematics and Statistics, an MSc in Medical Statistics, and a PhD in Epidemiology/Statistics. He has been a Royal Statistical Society accredited Chartered Statistician (CStat) since 2005.
His research is interdisciplinary, integrative and collaborative and has focused on using large-scale observational studies and randomized trials to generate reliable evidence for prevention of premature deaths and disability from chronic diseases. He co-leads the Statistical Group in the China Kadoorie Biobank (CKB) a large prospective study of 0.5M Chinese adults recruited from 10 regions (five urban and five rural) in China.
His work involves applying statistical, epidemiological, computational, and genetic tools to understand associations of exposures with chronic diseases, to drive improvements in population health via identification of novel treatment targets and implementation of precision strategies for primary and secondary prevention of major disease outcomes (such as cardiovascular disease, stroke, diabetes, and cancer).
He is responsible for developing and implementing a portfolio of CKB research themes (such Aging, Cardiovascular and Adiposity), ensuring robust, appropriate, and deliverable methodological input to study design, research conduct, and securing grant income as lead statistical co-investigator.
He is a co-lead for the Principles of Data Science module of the MSc in Global Health Science and Epidemiology and is leading the curriculum development for data science teaching, in addition to training and supervision of both undergraduate and postgraduate students (currently supervising three MSc projects and 8 DPhil projects).
He has also contributed chapters to four textbooks and was named as a Highly Cited Researcher for 2018 for highly cited papers that rank in the top 1% in his field of research. In 2022 he was listed amongst the top 1000 scientists in the UK in the Research.com Medicine rankings.
Jim Lewsey, PhD, CStat
University of Glasgow, Glasgow, United Kingdom
Jim Lewsey, PhD, is a reader in Medical Statistics and joined the University of Glasgow in 2007 having previously held posts at the London School of Hygiene and Tropical Medicine (2003-2007), University of Otago (2001-2003), Eastman Dental Institute-UCL (1998-2001), and University of Glasgow (1996-1998). He was awarded Chartered Statistician status from the Royal Statistical Society in 2010 and Chartered Scientist from the Science Council in 2012.
His personal research interests stem from methodological challenges faced when analyzing observational and experimental medical data and have included prognostic model development in the presence of missing data, continuous outcome monitoring of long-term outcomes, modelling dental caries data, and design of cluster randomized trials. His current methodological interests include multi-state survival analysis and modelling of data from natural experiments, and in his applied research he is developing a portfolio of alcohol research. Jim leads the Analysis of Linked Health Data (ALDA) program within HEHTA and is deputy lead of the IHW research theme 'Data Science - Using routine administrative data and record linkage for research'.
Jim is program director and teaches on the MSc in Health Technology Assessment. He also teaches and coordinates a medical statistics course on the Masters in Public Health.
8:00 - 17:00
Short Course Full Day Session
Reimbursement Systems for Pharmaceuticals in Europe
Level: Intermediate
Track: Health Policy & Regulatory
Separate registration required. Unlike marketing authorization for pharmaceuticals, mainly regulated at the European level by EMA, pricing and reimbursement decisions in Europe are managed by individual member states. Health care services are generally covered by a single public health insurer operating under the Ministry of Health supervision. As a monopoly buyer, this situation provides a leading position for the public health insurer to set reimbursement conditions. Therefore, based on each country’s set of regulations, processes, and values, wide variations exist in pricing and reimbursement decisions of pharmaceuticals. Using up-to-date governmental regulation sources and the ISPOR Global Health Care Systems Roadmap, this course will discuss health technology decision-making processes for reimbursement decisions for pharmaceuticals in France, Germany, Hungary, Italy, Poland, Spain, Sweden, and the UK. The course will describe these reimbursement systems, as well as compare, and bring into contrast their key characteristics.
This course is designed for individuals with intermediate experience within a single healthcare system wishing to broaden their appreciation of other reimbursement systems.
Faculty Member
Mondher Toumi, MD, PhD, MSc
Aix Marseille University, Marseille, France
Professor Mondher Toumi is an MD by training and holds two MSc in Biostatistics, and in Biological Sciences (option pharmacology) and a PhD in Economic Sciences. He is a professor of Public Health at Aix-Marseille University. After working for 12 years as a research manager in the Department of Pharmacology at the University of Marseille, he joined the Public Health Department in 1993. In 1995, he entered the pharmaceutical industry and worked there for 13 years.
Mondher Toumi was appointed global vice president at Lundbeck A/S in charge of health economics, outcome research, pricing, market access, epidemiology, risk management, governmental affairs, and competitive intelligence. In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in strategic decision-making.
In February 2009, he was appointed professor at Lyon I University in the Department of Decision Sciences and Health Policies. He launched the first European University Diploma of Market Access (EMAUD) an international course already followed by more than 500 students. Additionally, he recently created the Market Access Society to promote research and scientific activities around market access, public health and health economic assessment. He is chief editor of the Journal of Market Access and Health Policy (JMAHP).
Since September 2014, he joined the research unit EA3279 of the Public Health Department, at Aix-Marseille University (France) as Full Professor. Mondher Toumi is also visiting professor at Beijing University (Third Hospital).
In June 2022 Mondher Toumi founded InovIntell an international venture dedicated to AI in life sciences.
He is a recognized expert in health economics and an authority on market access and risk management. He published more than 200 scientific publications and authored or co-authored several books predominantly in the fields of market access and health economics.
12:00 - 13:00
Break
Lunch on own.
13:00 - 17:00
Short Course Afternoon Sessions
Early Health Technology Assessment
Level: Intermediate
Track: Health Technology Assessment
Separate registration required. This intermediate level course is for industry, researchers, advisory, regulatory, and funding agencies, governments, payers, clinicians, patients, and others who have a stake in improving care and services. The course focuses on Health Technology Assessment (HTA) concepts and methodologies in the early stages of product development (ie, early HTA). The research and development of a new medical technology is generally an expensive process; information about the potential clinical impact of a product—and its adoption and implementation into practice—at an early stage can justify the investment and guide further decision making all along the line. A major challenge is how to generate this information when no or only limited data is available.
This course provides an overview of the importance and constraints of early HTA, the evaluation and decision frameworks of different stakeholders, relevant research methodologies and the potential impact of the analyses. Using real-world case studies, we will present the process of working with stakeholders to frame the analysis, to gather evidence and to produce careful insights regarding potential new health technologies. Through a breakout exercise and group discussions, course participants will work on framing a product development decision and identify appropriate data collection and analysis methods.
PREREQUISITE: Attendance at short courses "Introduction to Health Technology Analysis", "Cost-Effectiveness Analysis Alongside Clinical Trials” and/or equivalent concepts and methodologies is prerequisite to attending this course.
Faculty Member
Janet Bouttell, MSc, PhD
Nottingham University Hospitals Trust, Nottingham, United Kingdom
Janet is a health economist at the University of Glasgow. From a background in chartered accountancy with KPMG, Janet joined the staff at the Health Economics and Health Technology Assessment (HEHTA) department in 2015. Janet completed her PhD in methods of Health Technology Assessment (HTA) for developers of diagnostic technologies in 2021. The PhD was funded by the Glasgow Molecular Pathology Node; an MRC funded collaboration between NHS Greater Glasgow and Clyde, the University of Glasgow and industry partners. Since 2021, Janet has worked on a Knowledge Transfer Partnership introducing health economics and business modelling concepts to a small developer of molecular pathology tests.
https://www.gla.ac.uk/researchinstitutes/healthwellbeing/staff/janetbouttell/
Sara Graziadio, PhD
York Health Economics Consortium, York, United Kingdom
Dr Sara Graziadio is a Project Director at YHEC since 2020. Sara has a PhD in Neuroscience and an MPhil in Statistics from the Newcastle University. She led systematic and pragmatic reviews to inform the design of clinical studies, for publications and health technology assessment submissions. She developed statistical and early economic models in multiple clinical areas and analysed care pathways. She has more than 15 years’ experience of analysis of complex qualitative and quantitative datasets and 10 years’ experience of leading successful projects.
Janneke Grutters, PhD
Radboud Institute for Health Sciences, Nijmegen, GE, Netherlands
Janneke Grutters, PhD, is associate professor at the department for Health Evidence, Radboud University Medical Center, the Netherlands. She holds an MSc in health sciences and a PhD in Health, Medicine, and Life Sciences, both from Maastricht University. She is a member of several scientific and societal committees and is initiator and chair of the HTAi Working Group on Early Health Technology Assessment.
Her key research interest lies in the early evaluation of innovations in healthcare. By assessing the (potential) value of an innovation in early stages of its development, she aims to contribute to more accountable decisions with regard to the development, research, and use of these innovations, leading to more efficient use of resources, and better healthcare. Learning from other fields of research, she develops, uses, and teaches methodology that enables such an early evaluation, and acknowledges all relevant uncertainties.
Ties Hoomans, PhD
London School of Economics and Political Science, London, United Kingdom
Ties has a background in in health economics and management science. His work focuses on the organization and delivery of health and social care services, with a particular interest in organizational behavior. Commonly combining quantitative and qualitative methods, Ties has done influential studies of the implementation and impact of new care approaches and guidance in everyday practice.
Through his research, Ties has contributed to methodology for studying complex interventions, including implementation strategies. He is experienced in engaging stakeholders in research processes, and works closely with local governments, care providers and other organizations within the health and care system.
Lotte Steuten, PhD
Office of Health Economics, London, Greater London county, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
Applied Cost-Effectiveness Modeling with R
Level: Intermediate
Track: Methodological & Statistical Research
Separate registration required. Historically, economic models for cost-effectiveness analyses have been developed with specialized commercial software (such as TreeAge) or more commonly with spreadsheet software (almost always Microsoft Excel). But more recently there has been increasing interest in using R and other programming languages for cost-effectiveness analysis which can offer advantages regarding the integration of input parameter estimation and model simulation, the evaluation of structural uncertainty, and the quantification of decision uncertainty, among others. Programming languages such as R also facilitate reproducibility of model-based cost-effectiveness analysis which is more relevant than ever given recent calls for increased transparency. While these tools are still relatively new, there is an increased interest in learning opportunities as evidenced by recent tutorials, workshops, and development of open-source software.
In this short course, participants will learn how to use R to develop a number of different types of economic models to perform cost-effectiveness analysis. Economic models will include time-homogeneous and time-inhomogeneous Markov cohort models, partitioned survival models, and semi-Markov individual patient simulations. The underlying assumptions of each model type will be summarized and the implementation in R will be presented in an accessible manner. Participants will be asked to modify the models in R (eg, adding health states, use of alternative time-to-event distributions) and run analyses (eg, cost-effectiveness analysis, probabilistic sensitivity analysis, evaluating structural uncertainty, and value of information analysis). To make this interactive aspect of the course as efficient as possible, all participants will have access to the GitHub repository prior to the course. It will contain R code to run the economic models and R Markdown files to explain and reproduce the analyses covered in the course. Participants who wish to gain hands-on experience are required to bring their laptops with R packages and scripts available. To get the most out of the course, it is important that registrants are able to do some R programming. Special instructions will be provided before the course.
Faculty Member
Devin Incerti, PhD
EntityRisk, Inc., San Francisco, CA, USA
Devin Incerti is the head of Data Science at EntityRisk with experience spanning health economics, biostatistics, and software engineering. Previously, Dr. Incerti was a Principal Data Scientist at Genentech working on statistical methodology for real-world and observational data. In his research, he developed approaches for causal inference in hybrid and external control studies and for prediction modeling with linked clinical and genomic data. Prior to Genentech, he was a senior economist at Precision Health Economics and the lead economist for the Open-Source Value Project at the Innovation and Value Initiative (IVI), where he performed research and developed software related to the value of medical technologies. He has developed software in a variety of languages (eg, Python, R, C++) and is an active contributor to the open source and health economics communities, including the development of hesim—a software tool for cost-effectiveness modeling and analysis. Dr. Incerti received BA degrees in Mathematics/Economics and Political Science from the University of California, San Diego, and a PhD in Public Policy from Princeton University.
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Valuation of Innovative Drugs
Level: Intermediate
Track: Health Policy & Regulatory
Separate registration required. The value of medical innovation depends on the perspective. Registration authorities (EMA, FDA) mainly consider the clinical value of the medical innovation, whereas national health authorities take a broader perspective by including clinical, economic criteria, and potential other criteria like equity and social values. Value-based pricing is the most widely accepted approach in the pricing and reimbursement process in Europe, which varies from the narrow concept based on the incremental cost-effectiveness ratio (ICER) threshold to broader approaches. Value-based pricing determines the maximum price from the national payer perspective. This price should exceed the minimum price for the investor acting in the international financial market, which is based on economic valuation theory. Finally, there are other stakeholders, eg, patients, physicians’ healthcare insurers, employers, with their specific assessment of the value of medical innovation varying from, respectively, quality of life, effectiveness, budget impact, and costs of lost productivity. This course offers an overview of the perspectives of the relevant stakeholders and their respective data requirements for value assessment of innovative drugs. The course will then describe in-depth description of the various value-based pricing methods, eg, ICER, multicriteria decision analysis (MCDA), comparative effectiveness research (CER), and relative effectiveness (RE). We include examples of orphan drugs and ATMPs which are most striking to illustrate the concepts, but we also include value assessment for more traditional innovative drugs in broad indications. Familiarity with health economic evaluation is desirable, but the course assumes little or no familiarity with economic valuation theory.
Faculty Member
Afschin Gandjour, MD, PhD, MA, MBA
Frankfurt School of Finance & Management, Frankfurt, Germany
Afschin Gandjour is a medical doctor, health economist, and philosopher. His research focuses on cost-effectiveness analysis, decision modeling, and value-based pricing of pharmaceuticals.
Gandjour received an MD from Hannover Medical School in Germany, an MBA from Duke University, a PhD in health economics from the University of Cologne in Germany, and an MA in philosophy from the University of Düsseldorf in Germany. He held faculty positions at the University of Cologne Medical School, Baylor College of Medicine, and Louisiana State University Pennington. In terms of research productivity, he ranks among the top professors in business administration in Germany (#20 in 2022 based on Wirtschaftswoche magazine).
Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics Institute, School of Pharmacy, University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
Dr. Garrison has worked in non-profit, industry, and academic settings. He received a PhD in Economics from Stanford University and has more than 200 publications in peer-reviewed journals.
Dr. Garrison was elected as ISPOR President for 2016-2017, and currently serves as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council. In September 2022, he was announced as the recipient of the 2022 Avedis Donabedian Outcomes Research Lifetime Achievement Award from ISPOR.
Marlene Gyldmark, MPhil
Idorsia Pharmaceuticals, Allschwill, BS, Switzerland
Marlene is a senior leader with several years’ experience in health service research, academia, and life science industry from both developing as well as developed countries.
Work experience includes building state-of-the art HEOR teams at Idorsia AG, Switzerland, at Roche Diabetes Care, Switzerland, and Roche Pharma global headquarters in Switzerland, as well as holding various positions within the field of Market Access, Pricing and HEOR at Roche Pharma, Novo Nordisk, and Pfizer, Denmark. Prior to the experience in industry, Marlene worked for more than 7 years in health service research in Denmark, Nepal and Tanzania.
In addition, Marlene also has many years of experience as an external lecturer in health economics at Copenhagen University, as well as leading an executive course in “Market Access for Pharmaceuticals” under Copenhagen University life-long learning.
Marlene’s interest from her early days at York University is to strengthen and support good decision making in healthcare and to foster collaboration and solutions across all stakeholders in the healthcare sector. Marlene is a member of the Board of Directors at ISPOR, and also supports the patient organization Institute of Neurodiversity (ION).
Marlene is educated as Cand Polit from Copenhagen University, and Health Economist from University of York, UK.
Mark Nuijten, MBA, PhD, MD
Ben Gurion University, Be'er Sheva, Israel
Mark Nuijten is a medical doctor, health economist, valuation economist, and healthcare publicist. He is a visiting professor at Ben-Gurion University in Israel, setting up the department on Clinical and Economic Valuation of Medical Innovation. He has become a leading health policy and economics expert over the last 2 decades, reflected in more than 200 publications and leading positions in scientific societies and editorial boards. Dr. Nuijten was board director of ISPOR (2002-2004) and chair of the Management Board of Value in Health (2002-2004). He was a member of the Editorial Advisory Board of Value in Health. He obtained his PhD in health economics (2003) on the thesis “In search for more confidence in health economic modelling” at the Erasmus University, Rotterdam.
Mark Nuijten is founder of A2M (Ars Accessus Medica) and founding partner of the Minerva International Health Economic Network. He was trained as a physician and worked in clinical research before obtaining his international MBA from Erasmus University, Rotterdam, where he later was a senior staff member. Prior to setting up Ars Accessus Medica, Dr. Nuijten was the founding managing director of the IQVIA Quintiles office in the Netherlands, which included European responsibility for the policy and health economic division.
He is a pioneer in the field of healthcare innovation in biotechnology and has been the first classical health economist successfully applying and developing Discounted Cash Flow methodologies for valuation of biotechnology innovation (eg, a pricing model to assess prices of expensive orphan drugs from an investor’s perspective—published in a Nature journal). He also developed an integrated valuation model, an interactive dynamic tool for the economic valuation of R&D projects, which can be used to optimize the initial clinical program (eg, indication, comparator, outcomes, and study design), and the associated pricing and market access pricing strategy.
Fred W. Sorenson, MSc
Cencora, Basel, Switzerland
Fred Sorenson is assistant director, Global Health Economics, Outcomes Research (HEOR) & Market Access at Xcenda, the consulting division of AmerisourceBergen where his work includes comparative effectiveness research, retrospective database studies, prospective studies and chart reviews, systematic literature reviews, global value dossiers and contributing to posters and publications. Mr. Sorenson also has experience, most predominantly in Europe, in developing strategy and gathering payer insights through payer interviews and advisory panels to inform HEOR initiatives and guide market access at the affiliate level. Mr. Sorenson has more than 35 years of experience in the healthcare industry, working in numerous therapeutic areas including cardiology, endocrinology, dermatology, oncology, neurology, autoimmune diseases, virology, and infectious disease.
Fred received his BS in both Psychology and Philosophy from the University of Southern Colorado and did his post-graduate studies in Sociology and Economics (master’s thesis on “Registration of Vital Birth Statistics in Nepal”) at the University of Basel in Switzerland.
Going Beyond the Standard: Exploring Advanced Survival Modeling Techniques
Level: Intermediate
Track: Methodological & Statistical Research
Separate registration required. Survival modeling techniques are commonly used to extrapolate clinical trial outcomes like overall survival to a time horizon that is appropriate for health economic evaluations. Standard parametric distributions, such as the exponential and Weibull, have been the de-facto standard for conducting such extrapolations but, with the advent of novel potentially curative therapies, these standard parametric distributions fail to capture the underlying survival trend. Newer techniques like response based landmark models, parametric mixture models, and mixture cure models provide novel ways to capture these more complex survival patterns. The purpose of this course is to enable participants to identify which methods are most appropriate in a specific context, considering underlying structural assumptions, and discuss how modeling choices propagate into health economic evaluations. To gain a more in-depth understanding of the impact of the choice for a specific method, there will be walkthroughs of exercises which participants will be able to practice in their own time.
Faculty Member
Elisabeth Fenwick, PhD
OPEN Health Evidence & Access, Oxford, OXF, United Kingdom
Elisabeth Fenwick is Deputy Chief Scientific Officer in Evidence & Access at Open Health, based in Oxford in the UK.
Liz provides scientific and strategic support to HE projects globally. She has extensive experience in economic evaluation and health economic modeling having worked in the field for over 20 years. She has worked on a variety of projects in a wide range of disease areas including oncology, respiratory, infectious diseases, cardiology, ophthalmology, and orphan diseases.
Liz has also contributed to methods in the field, in particular relating to decision analytic modeling and simulation methods, probabilistic decision analytic modeling and value of information analysis. Liz was a member of the ISPOR joint task force on good research practices in modeling and a co-author on the joint taskforce paper on uncertainty and co-chaired/co-authored the recent ISPOR task force assessing emerging good practice in value of information analysis for research decisions.
Liz has a PhD and MSc in Health Economics as well as an MSc in Operations Research and joined Open Health from ICON plc where she led the modeling team for the global HE group. Prior to her consultancy career, Liz spent over 15 years as an academic working at University of York, McMaster University, and most recently University of Glasgow.
Sven Klijn, MSc
Bristol-Myers Squibb, Utrecht, ZH, Netherlands
Sven Klijn is director at Bristol Myers Squibb in the HEOR Economic & Predictive Modeling group, where he leads the innovative modeling agenda in hematology. In addition, Sven has an active role in providing modeling education and masterclasses at international congresses. He has widely published on innovative methods, especially in the field of survival extrapolation. Sven has a training in public health and health economics and previously had various roles in CROs related to health-economic modeling.
Claire Simons, PhD, MSc, MMATH
OPEN Health Group, York, NYK, United Kingdom
The Role of Digital Endpoints in the Value Generation for Health Technologies
Level: Intermediate
Track: Health Policy & Regulatory
In this short course, participants will learn about new endeavors for the value generation in the patient's centric digital era. With the exponential growth of the market of digital health with digital therapeutics, digital sensors, or mobile app we can understand better the unmet patients’ needs and identify new opportunities for healthcare system’s efficiency gains. The plethora of new digital endpoints enters clinical trials arena and helps to enrich the value propositions for medicines. It is the highest time for HEOR to expand its frontier to capture these new opportunities with growing variety and veracity of digital data from the patient’s perspective. Participants will first be familiarized with the key changes in the healthcare system in the digital era and then learn how to utilize the digital endpoints in the process of the clinical and economic value generation while ensuring the methodological rigor required for evidence-based decision making with a patient-centric approach.
Participants who wish to gain hands-on experience must bring personal laptop.
PREREQUISTE: Participants should possess the basic skills and understanding of cost effectiveness and budget impact models.
Faculty Member
Jochen Klucken, MD
University of Luxembourg, Luxembourg, N/A, Luxembourg
Prof. Dr. Med. Jochen Klucken is a neurologist, neuroscientist, full professor and the FNR-PEARL chair of Digital Medicine and head of the Digital Medicine research group (dMed) at the LCSB (Luxembourg Center for Systems Biomedicine) of the University of Luxembourg, the LIH (Luxembourg Institute of Health) and the CHL (Centre Hospitalier de Luxembourg). Digital transformation in medicine utilizes better objective real-world data and smarter algorithms ("artificial intelligence") tailored to patients’ and healthcare providers’ needs and requires a profound understanding of data-driven medicine (procedures) integrated into existing medical workflows and patients’ everyday life behaviour and its disease-dependent limitation.
Translational Research in Digital Medicine includes engineering and data-research, IT-requirements, but also clinical knowledge, societal expectations in ethics, acceptance, and regulation in order to enlighten the abilities of digitalization in medicine, and to avoid the downsides coming with an unheard level of transparency. The dMed research focuses on 1) shaping and innovating personalized digital healthcare solutions, and 2) evaluating the medico-socio-economic benefit of new digital medical devices and services.
Prof. Klucken is also co-leading the EU Taskforce for Harmonized Evaluation of Digital Medical Devices with a strong focus on Digital Medical Devices and their innovative clinical evaluation frameworks. Future digital medicine ecosystems will provide real-world data for continuous quality monitoring of existing healthcare procedures and innovative evaluation concepts providing real-world evidence for digital medicine.
Katarzyna Kolasa, PhD
Kozminski University, Warsaw, MZ, Poland
Katarzyna Kolasa PhD
Professor of Health Economics at Kozminski University Warsaw Poland
Digital Health Start Me Up Course leader
ISPOR Digital Health SIG founder
“The Digital Transformation of the Healthcare System” Routledge; 1st edition (July 31, 2023)
Driven with passion to improve healthcare, Katarzyna has focused her academic and busi-ness career on the health economics.
She has been working with multiple pricing & reimbursement challenges worldwide for the last 25 years, while holding various Regional and Global leadership positions at Astra Zeneca, BMS, Biogen Idec, Lundbeck, GE Healthcare, Straub Medical, BD and the Swedish county council of Kalmar. Katarzyna is mentor and consultant to start ups involved in the development of innovative medical devices and digital health solutions from both Holland and Poland.
Since 2000 she has been academic teacher and supervisor for over 30 MBA and Phd stu-dents. In partnership with the Polish Medical Research Agency, Deloitte Digital and the Polish Central Hospital of Ministry of Interior Affairs she founded the first Digital Health six months educational program designed for digital transformation leaders working in the healthcare sector. Katarzyna developed innovative Master Program Health Economics & Big Data (HEBDA) which first edition was financed by EU Power Grant 2018 as well.
She is the founder of the Global Special Interest Group Digital Health and short courses “The Role of Digital Endpoints in the Value Generation for Health Technologies”, “Risk-Sharing/Performance-Based Arrangements in Developing Countries” at International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She is the current member of Edu-cational and post member of Health Science Policy Councils at ISPOR as well.
Katarzyna has dedicated her academic research towards the methodological advancements into the value assessment of pharmaceuticals, medical devices, and digital health solutions. Passionate about Big Data, she led the first project of machine learning adaptation for the optimal utilization of CT scanners granted by the Polish Ministry of Health as well. Since January 2022, she is the leader of AI special interest group at the Polish Chamber of Physi-cians. With the patronage of the Polish Parliamentary Commission for Innovation & Digitali-zation, she organized the first dialog about the societal preferences towards the adoption of AI in the healthcare in Poland.
Being coauthor of more than 50 IF publications, she presented her research at more than 60 international scientific conferences. As of 2022, Google Scholar reports over 730 cita-tions to her work.
Alison Ritchie, PhD
Parexel, Gamston, England, United Kingdom
Alison is the global head of Sensor Solutions at Parexel, and also supports real-world data sales and operational delivery. Alison has worked at Parexel for over 20 years and has over 24 years’ experience within the clinical trial industry. She also plays a key role by selecting and implementing real-world data and connected sensor device solutions for clinical trial use.
Alison has a broad range of expertise within the technology area of Clinical Trials phase 1-4, with a background within multiple technology areas, including operations, product delivery and client management. She is also passionate about diversity, equity and inclusion whilst advancing technology within Clinical Trials and as a result advancing patients care and acts within the Parexel Committee, as well as being an active enterprise advisor.
Reporting Economic Evaluations: How to Use CHEERS 2022
Level: Introductory
Track: Economic Evaluation
Separate registration required. Economic evaluations make up a large proportion of the literature in health economics and outcomes research. These studies are increasingly used in healthcare decision making and it is critical that they are reported correctly. This course is intended to familiarize participants with the rational for, and content of, the important reporting items in economic evaluations. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) are an EQUATOR guideline and represent the state of art for reporting economic evaluations. CHEERS consists of 28 reporting items relating to the introduction and design of economic evaluations, their methods, and the reporting of results. The rationale for the various items will be given, along with illustrative examples explaining their content. Participants will learn how to use CHEERS in reporting their own economic evaluations, and in reading evaluations conducted by others. In addition, key issues relating to the use of CHEERS will be discussed.
Faculty Member
Federico Augustovski, MD, MSc, PhD
Institute for Clinical Effectiveness and Health Policy (IECS), Buenos Aires, B, Argentina
Federico Augustovski is the current director of Health Economic Evaluations and Technology Assessment at the Institute for Clinical Effectiveness and Health Policy (IECS), an independent non-profit organization affiliated to the University of Buenos Aires, a CONICET (National Scientific and Technical Research Council) center, and one of the few INAHTA Health Technology Assessments agencies in Latin America. He is the director of the WHO Collaborating Centre in Health Technology Assessment and Economic Evaluations at IECS. He is a professor of Public Health at the School of Public Health of the University of Buenos Aires, where he teaches courses for graduate and postgraduate students in Decision Sciences; Patient Reported Outcomes (PRO) Development in Health as well as Health Economic Evaluations.
He was elected president 2017-2020 of ISPOR (The Professional Society for Health Economics and Outcomes Research). He is also the founding editor-in-chief for Latin America of Value in Health Regional Issues (ViHRI), the ISPOR peer-reviewed journal for Latin America, Asia, and Central & Eastern Europe and Africa. He is the director of the PAHO affiliated PROVAC Center of Excellence for decision making in vaccines. He leads a multidisciplinary team devoted to clinical and economic evaluations of new and existing preventive, diagnostic and therapeutic technologies that works doing research, education, and technical support with public and private health decision makers in Latin America.
He got his medical degree with honors at the University of Buenos Aires (1986-1991), and he is a specialist in family medicine. He practiced family medicine and was a staff physician for more than 20 years at the Family and Community Medicine Division of the Hospital Italiano de Buenos Aires (the leading academic non-profit hospital in Argentina). He got his MSc in Epidemiology (Harvard School of Public Health, 1997-1999). He was an Alban Scholar of the European Union in Health Economics (2003-2004) getting research and training experience at the Centre for Health Economics at York University in the UK.
His research production and publication in international peer-reviewed journals (with more than 160 PubMed indexed papers), concentrates on health technology assessments, health economic evaluations (ie, multi-country studies in Latin America, both model-based and individual patient level piggyback studies, PRO and preference status measures and validation (ie, derivation of Argentine, Uruguay and Peruvian weights for the EQ-5D, Argentine SF-36 validation, and discrete choice experiments).
Nathorn Chaiyakunapruk, PharmD, PhD
Department of Pharmacotherapy, College of Pharmacy, University of Utah, Salt Lake City, UT, USA
Dr.Chaiyakunapruk (Or Nui) is a Professor in the College of Pharmacy at The University of Utah. earned his PharmD from University of Wisconsin-Madison and PhD from University of Washington. His expertise is in Health Technology Assessment. He applied his methods in health economics, real world data analysis, and evidence synthesis to support national and global policy, especially his contributions to the World Health Organization and Centers for Disease Control. His current research works have been focused on health economics and health equity. He is a co-author of CHEERS 2022 (Consolidated Health Economic Evaluation Reporting Standards Statement) and WHO guide for standardization of economic evaluations of immunization programmes, 2nd edition. Dr.Chaiyakunapruk has an H-index of 55 and is an author of more than 300 international publications in peer-reviewed journals.
Michael Drummond, PhD
University of York, Lichfield, Staffordshire, United Kingdom
Michael Drummond, BSc, MCom, DPhil is professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 700 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Budget Impact Analysis II: Applications and Design Issues
Level: Intermediate
Track: Economic Evaluation
Separate registration required. This course covers the concrete application of the 6-step approach for developing budget impact analyses and provides hands-on learning with two different budget impact models programmed in Excel. The course will review the basics of budget impact analysis, interpretation of results, simplicity versus accuracy and face validity, and how budget impact analyses are used by payers and other decision makers. Technical topics will include static versus dynamic budget impact models, considerations for device and diagnostic technologies, and realistic features such as patient copayments and use of generics. The instructors will walk through 2 different budget impact analyses programmed in Excel (one static and one dynamic) and work with participants during hands-on exercises to enhance these models. The instructors will also review good practices for building budget impact models and provide a number of Excel tips. The Excel-based budget impact models used for the course will be provided to participants in advance of the presentation. This course is designed for those who have basic knowledge of budget impact analyses and desire exposure to these analyses in Excel. Participants who wish to gain hands-on experience must have Microsoft Excel for Windows installed on their computers.
Faculty Member
Thor-Henrik Brodtkorb, PhD
RTI Health Solutions, Ljungskile, O, Sweden
Thor-Henrik Brodtkorb, PhD, is executive director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 12 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision-analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and ISPOR.
At RTI-HS, Dr. Brodtkorb leads the development of cost-effectiveness, cost-utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Anita Brogan, PhD, MSc
RTI Health Solutions, San Diego, CA, USA
Anita Brogan is vice president of Decision Analytic Modeling within the Health Economics division at RTI Health Solutions (RTI-HS). She holds a PhD in Operations Research from the University of North Carolina and has been with RTI-HS for 20 years. She has presented workshops and short courses on decision-analytic modeling techniques in a variety of venues, including meetings of ISPOR and the Academy of Managed Care Pharmacy (AMCP). Dr. Brogan has held an adjunct professorship in the Fermanian School of Business at Point Loma Nazarene University. She is also a co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
In her role at RTI-HS, Dr. Brogan uses analytical techniques to assess and present the health and economic impact of emerging pharmaceutical and biotechnology interventions. She leads development of user-friendly and transparent cost-effectiveness, cost-utility, and cost-consequence models; budget-impact models; resource allocation models; and infectious disease transmission models programmed in Microsoft Excel and other platforms. She has experience with numerous types of modeling procedures, including Markov and other stochastic models, simulation, regression, linear and nonlinear programming, and various types of sensitivity analysis. Dr. Brogan has developed models and analyses in the areas of HIV, hepatitis C, RSV, Ebola, influenza, cystic fibrosis, bone health, mental health, women’s health, oncology, osteoporosis, chronic pain, age-related macular degeneration, hospital-acquired infection, financial portfolio optimization, and vehicle routing. Her research has been presented at various professional conferences and published in peer-reviewed journals.
Ashley Davis, PhD, MSc
RTI Health Solutions, Research Triangle Park, NC, USA
Ashley Davis is a senior director within the Health Economics division at RTI Health Solutions (RTI-HS). She holds a PhD in Industrial Engineering and Management Sciences from Northwestern University and has been with RTI-HS for 10 years. She has presented short courses on budget impact modeling techniques in a variety of venues, including meetings of The Professional Society for Health Economics and Outcomes Research (ISPOR).
In her role at RTI-HS, Dr. Davis uses analytical methodologies to evaluate the clinical and economic value of upcoming pharmaceutical products and changes to healthcare policies. She has developed user-friendly and transparent cost-effectiveness, cost-utility, and cost-consequence models; budget-impact models; resource allocation models; and infectious disease transmission models programmed in Microsoft Excel and other platforms. She has experience with numerous types of modeling techniques, including Markov and other stochastic models, simulation, statistical analysis, linear and nonlinear programming, robust optimization, and various types of sensitivity analysis. Dr. Davis has developed models and analyses in the areas of HIV, hepatitis C, cystic fibrosis, herpes zoster, influenza, pneumococcal disease, respiratory syncytial virus, severe asthma, chronic obstructive pulmonary disease, eosinophilic esophagitis, spinal surgery, cytomegalovirus, and organ transplantation. Her research has been presented at various professional conferences and published in peer-reviewed journals.
Stephanie Earnshaw, PhD, MS
RTI Health Solutions, Pittsboro, NC, USA
Stephanie Earnshaw is senior vice president of Health Economics at RTI Health Solutions (RTI HS). She received her PhD in Industrial Engineering at North Carolina State University and has performed health outcomes and health services research for over 30 years, 24 of which have been with RTI-HS. She has presented workshops and various courses on decision-analytic modeling techniques for pharmaceutical companies and organizations such as ISPOR, the Academy of Managed Care Pharmacy (AMCP), and the Centers for Disease Control and Prevention (CDC). Dr. Earnshaw has served on the ISPOR Board of Directors and as Chair of the Audit Committee and is the current Educational Council Chair. She has held an Adjunct Faculty appointment at the University of North Carolina’s Eshelman School of Pharmacy, Division of Pharmaceutical Outcomes and Policy, is honored as a Distinguished Alumni in Industrial and Systems Engineering at North Carolina State University and is one of the lead authors of “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Dr. Earnshaw’s research focus is in applying decision-analysis techniques to industry-related issues and health care problems. Her areas of specialization include mathematical programming (constrained optimization), network optimization, and Markov, simulation, and other state transition modeling. In addition to developing budget-impact and cost-effectiveness models to support health technologies for the pharmaceutical, biotechnology, and diagnostic and medical device industry, she has developed innovative mathematical models using these methods to determine pricing strategy, predict clinical outcomes, allocate resources, and cost care pathways particularly in support of medical diagnostics. Therapeutic areas include cardiovascular disease, gastrointestinal disorders, respiratory disease, acute care, infectious disease, osteoporosis, vaccines, and oncology. She is a member of ISPOR and the Institute for Operations Research and the Management Sciences. She has presented her work at professional conferences and has published in several peer-reviewed journals.
Unveiling the EU-HTA Regulation: A Deep Dive Into the Joint Clinical Assessment (JCA)
Level: Intermediate
Track: Health Policy & Regulatory
Separate registration required. In January 2022, a significant milestone was reached in the field of Health Technology Assessment (HTA) with the enforcement of the EU-HTA regulation. This regulation aims to harmonize the HTA process across the EU and improve efficiency by reducing duplication of efforts for national HTA authorities. Replacing the existing voluntary network of national HTA agencies and the EU-funded Joint Actions (EUnetHTA), the EU-HTA regulation introduces a permanent framework for collaborative work. To this end, it covers horizon scanning, joint scientific consultations, joint clinical assessments, and voluntary cooperation on non-clinical aspects of HTA which remains a national responsibility. From 2025 onwards, the regulation will be gradually implemented, starting with cancer treatments and advanced therapy medicinal products (ATMPs), followed by orphan drugs in 2028 and all other drugs in 2030. In this interactive course, we will discuss the methodological deliverables, procedural guidelines and templates produced by EUnetHTA 21, a consortium of EU-HTA agencies in charge of the key deliverables. We will focus on the latest developments around the joint clinical assessment, aiming to produce one single assessment of the relative clinical benefit of the new drug that is the basis for all EU member states. This assessment starts with a survey among the member states asking for the patient/population, intervention, comparison and outcomes (PICOs) they need. The course will also address the challenges associated with the position of the joint clinical assessment alongside the EMA assessment and the national market access processes, and the involvement of manufacturers and patient representatives.
In the final segment of this course, we will examine the potential implications and future prospects of the EU-HTA regulation. We will discuss how this regulation could potentially reshape the landscape of HTA in the EU, and what this means for stakeholders, including healthcare providers, patients, and pharmaceutical companies. We will also consider the potential challenges and opportunities that may arise as the regulation is progressively implemented. This will include a discussion on how to navigate the transition period and how to prepare for the full implementation of the regulation.
To ensure a practical understanding of these concepts, we will divide the classroom into smaller groups for a portion of the course. These groups will work on a practical exercise involving a hypothetical cell or gene therapy, using evidence from single-arm studies. By the end of this course, participants will have gained a comprehensive understanding of the EU-HTA regulation and will be equipped with the knowledge and tools to navigate this new era of health technology assessment in the EU. Join us as we unravel the complexities of the EU-HTA regulation and its impact on the future of HTA in the EU.
PREREQUISTE: Participants who wish to gain hands-on experience are required to bring their laptops.
Faculty Member
Karin Becker, MSc, PhD
Boehringer Ingelheim GmbH, Ingelheim, Germany
Maureen Rutten-van Mölken, PhD
Erasmus University Rotterdam, Erasmus School of Health Policy and Management (ESHPM) and Institute for Medical Technology Assessment (IMTA), Rotterdam, Netherlands
Maureen Rutten-van Mölken is full professor of Economic Evaluation of Innovations for Health at the Erasmus School of Health Policy & Management, where she is head of the Health Technology Assessment department. She is also scientific director of the Institute for Medical Technology Assessment, a company fully owned by the Erasmus University Holding. She holds an MSc in health sciences and a PhD in health economics, both from the University of Maastricht. She teaches Health Technology Assessment and Pharmaceutical Pricing and Market Access in the Master-program Health Economics Policy and Law and in the European Master in Health Economics and Management.Her expertise includes cost-effectiveness analysis alongside clinical trials and observational studies, probabilistic decision analytic modeling, health economic outcomes research and innovative payment models. She worked in many disease areas, including respiratory diseases (COPD, asthma), rheumatology, diabetes, cardiovascular disease, and Pompe disease. She has a special interest in the cost-effectiveness, and financing and payment of complex multi-faceted interventions that involve system-level changes (e.g., integrated care programs, disease management programs, hospital at home programs, outpatient rehabilitation programs, remote patient monitoring). She also has an interest in the health economics of personalized and precision medicine. She was the coordinator of SELFIE, a Horizon2020 project on multi-criteria decision analysis of integrated care programmes for patients with multi-morbidity. She was a principal investigator in Horizon2020 project HEcoPerMed that developed guidance on health economic modelling of personalised medicine and applied this guidance to various pharmacogenetic interventions. She also coordinates the Medical Delta program From Prototype to Payment.
Christophe Sauboin, MSc
Boehringer Ingelheim GmbH, Ingelheim, Germany
Frederick Thielen, PhD
Erasmus University Rotterdam, Erasmus School of Health Policy and Management (ESHPM), Rotterdam, Netherlands
Causal Inference and Causal Diagrams in Big, Real-World Observational Data and Pragmatic Trials
Level: Experienced
Track: Real World Data & Information Systems
Separate registration required. Innovative causal inference methods are needed for the design and analysis of big real-world observational data and pragmatic trials used for outcomes research and health technology assessment. This course will introduce the principles of causation in comparative effectiveness research, the use of causal diagrams (directed acyclic graphs; DAGs) and focus on causal inference methods for time-independent confounding (multivariate regression, propensity scores) and time-dependent confounding (g-formula, marginal structural models with inverse probability of treatment weighting, and structural nested models with g-estimation). The “target trial” concept and a counterfactual approach with “cloning – censoring – weighting” will be used to apply causal methods to big real-world datasets with case examples from oncology, cardiovascular disease, HIV, nutrition and obstetrics. The course consists of lectures, practical training exercises, case examples drawn from the published literature which will be used to comprehend the use of causal inference in health technology assessment bodies (eg, NICE , IQWiG), and an interactive discussion with Q&A. The intended audience includes all stakeholders in medical and public health decision making and healthcare, and researchers from all substance matter fields, statisticians, epidemiologists, outcome researchers, health economists, modelers and health policy decision makers interested either in methods of causal analysis or causal interpretation of results based on the underlying method. Course material includes all session handouts, exercises with solutions, a comprehensive background reading library, and software recommendations.
PREREQUISITE: Students are expected to have a basic knowledge in epidemiologic studies and methods (including the concept of confounding).
Faculty Member
Douglas E. Faries, PhD
Eli Lilly and Company, Indianapolis, IN, USA
Doug Faries graduated from Oklahoma State University with a PhD in Statistics in 1990 and joined Eli Lilly and Company later that year. Doug’s early research interests included development and implementation of novel clinical trial designs to improve and accelerate decision making for drug development and neuroscience clinical trials. Over the past 15 years, Doug has focused his research interests on statistical methodology for real world data. Specific research interests include causal inference, unmeasured confounding, and use of real world data for personalized medicine. Currently, Doug is a research fellow at Eli Lilly, leading the Real World Analytics team. In this role, Doug is involved with the design and analysis of observational research including comparative effectiveness analyses and the development of real world analytical capabilities.
He has authored or co-authored over 100 peer-reviewed manuscripts, edited a textbook “Analysis of Observational Healthcare Data Using SAS”, and is active in the statistical community as a publication reviewer, speaker, workshop organizer, in industry working groups, and teaching short courses.
Felicitas Kuehne, MSc
Pfizer Pharma GmbH, Berlin, 0, Germany
Felicitas Kuehne is an outcomes research manager at Pfizer Pharma GmbH in Germany. She is conducting epidemiological, economic, and health service research using real-world data as well as decision modelling studies. Further, Felicitas is a senior scientist in decision-analytic modeling and epidemiology at the Department of Public Health, Health Services Research and Health Technology Assessment at UMIT in Hall i.T., Austria. She is leading the Program on Causal Inference in Science and is the Coordinator of the HTADS course “Causal Inference for Assessing Effectiveness in Real World Data and Clinical Trials: A Practical Hands-on Workshop”. At the Institute, she conducts decision-analytics as well as epidemiological studies in several disease areas and teaches epidemiology classes at several universities in Europe as well as at international conferences.
Felicitas completed her Master of Science in Health Policy and Management at the Harvard School of Public Health in 2001. From 2001 to 2011 she worked as a consultant for pharmaceutical companies, conducting several cost-effectiveness analyses in a variety of disease areas. She joined UMIT in 2011 and Pfizer Pharma in 2022.
Her research interests include evaluating public health intervention by applying evidence-based quantitative methods from epidemiology, comparative effectiveness research, health services and outcomes research, economic evaluation, and decision sciences. Her current substantive research focuses on identifying synergies of causal inference and decision science. The main disease areas are pneumococcal disease, cardiovascular diseases, cancer, and infectious diseases including HIV/AIDS and hepatitis C.
Her studies at Harvard were partly sponsored by complementary scholarships of the Carl Duisberg Gesellschaft (1 year) and the Boehringer Ingelheim Stiftung (2 years). In 2013, Felicitas received with her team the third German research award for general medicine of the Dr. Lothar Beyer Foundation with a shared decision-making project in the cardiovascular disease area. Felicitas Kuehne was awarded three Tyrolean Science Fund (TWF) grants in 2016, 2020 and 2022 for causal inference projects.
Uwe Siebert, MD, MPH, MSc, ScD
UMIT TIROL - University for Health Sciences and Technology, Hall in Tirol, Austria. ONCOTYROL - Center for Personalized Cancer Medicine, Innsbruck, Austria. Harvard T.H. Chan School of Public Health and Harvard Medical School, Hall in Tirol, 7, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a Professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), Chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL - University for Health Sciences and Technology in Austria and Director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also Adjunct Professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in Decision Sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE, EUCAPA, PREMIO COLLAB). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (>30,000 citations, H index >80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
17:30 - 18:30
Educational Symposia
Is There Any Hope for the Value of Hope?
Digital Conference Pass
The landscape for innovative pharmaceutical interventions is rapidly evolving which is translating to hope in disease areas where there is huge unmet need. To continue incentivising innovation the corresponding policy should be reviewed and updated, integrating the perspective of patients who ultimately benefit. The ‘Value of Hope’ as an important value element of new treatments and has generally been supported as a concept in literature to date, however, HTA policy assessment frameworks are less supporting and seem to be lagging in producing a standardised method to incorporate a wider perspective including Value of Hope. The aim of the symposium is to engage in a thought provoking and insightful conversation around current perceptions towards Value of Hope.
Value of Hope will be introduced as a relevant concept for HTA decision making in the era of innovative new therapies and the current landscape and gaps will be highlighted. The topic will be debated from a range of viewpoints including: a proponent of Value of Hope, an industry representative, a patient advocate and a HTA perspective. The symposium discussion, guided by the moderator, will confer ways to empirically measure hope including the practicality of those that have been researched and lesser explored methods. Novel policy concepts to integrate Value of Hope into HTA decision-making, without completely overturning current practices will be deliberated. The panellists will also discuss the relative importance of Value of Hope as a value element and consider the benefits, challenges, and controversies of incorporating it into a HTA framework.
Sponsored by Putnam Inizio Advisory
Sponsor
Putnam Inizio Advisory
Moderators
Louise Longworth, PhD
Putnam PHMR Ltd., London, United Kingdom
Louise Longworth is a health economist specialising in HTA methods and processes, and the measurement and valuation of health. In her role at Putnam PHMR, Louise led its HEOR activities and numerous HTA submissions. She has previously worked in academia and with NICE in the UK, including in its Technology Appraisals programme, conducting independent evidence reviews and as a member of its Technology Appraisal Committee. She is a member of the EuroQol Group, the group responsible for the development of the EQ-5D, and has held positions on its Board of Directors and Executive Committee.
Speakers
Meindert Boysen, PharmD, MSc
Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Meindert is responsible at NICE for international partnership working, developing NICE’s role in international fora and representing NICE at key international meetings, further sustaining and developing NICE’s international reputation. Until recently, Meindert was the director at NICE responsible for delivery of the programmes that together form the centre for health technology evaluation. Including the technology appraisals, highly specialised technologies, medical technologies evaluation, diagnostic assessment and interventional procedures programmes. After qualifying as a pharmacist from Utrecht University, he worked in a hospital in the Netherlands, and held positions in health outcomes and sales in the pharmaceutical industry. Meindert completed the MSc in Health Policy Planning and Financing at the London School of Hygiene and Tropical Medicine and the London School of Economics & Political Sciences and briefly worked for the King’s Fund before starting at NICE in 2004. Meindert was a member of the Board of NICE and a director of the board of the International Society for Pharmacoeconomics and Outcomes Research (2017-2020).
Lou Garrison, PhD
The Comparative Health Outcomes, Policy, and Economics Institute, School of Pharmacy, University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
Dr. Garrison has worked in non-profit, industry, and academic settings. He received a PhD in Economics from Stanford University and has more than 200 publications in peer-reviewed journals.
Dr. Garrison was elected as ISPOR President for 2016-2017, and currently serves as co-chair of ISPOR’s Policy Outlook Committee for the Health Science Policy Council. In September 2022, he was announced as the recipient of the 2022 Avedis Donabedian Outcomes Research Lifetime Achievement Award from ISPOR.
James Ryan, MSc
AstraZeneca, Cambridge, CAM, United Kingdom
James represents AstraZeneca on regional and global HTA Policy around the world and is a co-lead on EFPIA’s HTA Working Group’s methodology workstream on the EU HTA Regulation. With over 25 years of experience in health economics and payer evidence generation, James has extensive knowledge and experience of making HTA submissions across Europe and beyond. In addition to his policy role, he also leads the oncology global HTA and Modelling Science team at AstraZeneca.
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
Dr. Ryll holds a medical degree from the Free University of Berlin, Germany, and a PhD in Biomedical Sciences from University College London, UK. After losing her husband to Melanoma, she founded the Melanoma Patient Network Europe and developed a special interest in patient-centric clinical research and innovation, innovative trial designs and novel drug development concepts. Between 2019 and 2021, she was member of the first EU Cancer Mission Board where her particular interest has been how to best leverage the potential of personalized medicine for patients and society through novel forms of collaboration and the support of health policy and governance. Currently, she works as strategist at SIR, Stockholm School of Economics' Research Institute for Mission Zero Cancer, an innovation ecosystem in health financed by Vinnova, Sweden's innovation agency.
Mon 13 Nov
7:00 - 17:00
Registration Hours
7:30 - 8:30
Morning Coffee Service
8:30 - 9:45
Plenary Session
European Health Data Space – RWE Put to Work for Public Health
Digital Conference Pass
Establishing a European Health Data Space (EHDS) giving citizens easy access to their own health care records also holds a potential for RWE generations via secondary use of such data. The session will explore the political vision and realities of EHDS including discussion and challenges related to any opt-in or opt-out options. We will hear patients’ views both regarding the utility of an EHDS as well as issues related to data privacy. Pathfinder projects like Towards European Health Data Space, TEHDAS, will shed light on the challenges with bringing numerous and often diverse data sources together on one platform, and the European Medicines Agency will present their vision and concrete examples of RWE in the regulatory decision-making process.
Moderators
Steffen Thirstrup, MD, PhD, CMO
European Medicines Agency, Amsterdam, Netherlands
Steffen Thirstrup is a medical doctor and board-certified specialist in clinical pharmacology and therapeutics. He holds a PhD in pharmacology and has a long background in clinical internal medicine with special emphasis on adult respiratory medicine. Additionally, Dr. Thirstrup was appointed adjunct professor in pharmacotherapy at the Faculty of Health Sciences, University of Copenhagen, in 2012.
From 2004-09 Steffen Thirstrup worked at Danish Medicines Agency first as the Danish member of CHMP at the European Medicines Agency (EMA) for five years including 10 months as joint CHMP- and CAT-member, followed by a short period as head of Danish Institute for Rational Pharmacotherapy dealing with HTA and best practice guidelines for primary care. In 2011 Prof. Thirstrup rejoined the licensing division at the Danish Medicines Agency acting as Head of Division for Medicines
Assessment and Clinical Trials. During this period Prof Thirstrup co-chaired the European Commission’s working group on market access for biosimilars medicinal products and acted as key scientific contact for the managing entity of the IMI beneficiaries for the PROTECT collaboration (Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium).
In March 2013, Prof Thirstrup joined the pharmaceutical consultancy company NDA Group AB as a full- time medical advisor on NDA’s regulatory advisory board. In April 2014 Prof Thirstrup was appointed as director for the Regulatory Advisory Board at NDA Regulatory Services Ltd.
Since June 2022 Prof Thirstrup has been the Chief Medical Officer at the European Medicines Agency, Amsterdam, The Netherlands
Speakers
Markus Kalliola, MSc, MSocSc
The Finnish Innovation Fund Sitra, Helsinki, Finland
Markus Kalliola is the project director in Health data 2030 project in The Finnish Innovation Fund Sitra and the coordinator of Joint Action Towards The European Health Data Space (TEHDAS). Prior to Sitra he worked in the European Commission in DG Health and food safety where he was responsible of the cross-border healthcare IT projects. Mr. Kalliola has vast experience in data economy and in the next years he will work towards healthier and fairer data economy for Europe as well as towards more competitive health data ecosystem in Finland.
Mr. Kalliola holds a positions of trust in
• HMA-EMA joint big data steering group,
• HMA-EMA Darwin EU advisory board
• DNV Digital health advisory board
• EDAH advisory board
• HealthHub Finland EDIH advisory board
• Steering group for the secondary use of health data in Finland
Trine Pilgaard, MSc
Pfizer, Ballerup, 84, Denmark
Trine Pilgaard, Director, Market Access at Pfizer Aps. is a Global Market Access Leader with more than 15 years of experience working in the pharmaceutical industry. Trine started her career at the World Health Organization, working in the pharmaceutical department, where she co-led and co-authored the EU-funded Pharmaceutical Pricing & Information Project (PPRI). She then moved to Novo Nordisk, where she had various roles within Global Market Access, amongst others, working to support the launch of new assets within hemophilia. After Novo Nordisk, Trine joined the Global Leadership team at Lundbeck, responsible for launching their new schizophrenia product. Today, Trine heads-up Pfizer Aps’s Market Access department, ensuring access and reimbursement of Pfizer’s products in Denmark & Iceland. Trine’s department is responsible for Health Technology Assessments, Health Economics, Market Access, Outcomes Research, Real-World Evidence, pricing, and tenders. In addition to her role as Director, Market Access at Pfizer, Trine is active in the Danish Trade Association and part of the committee of Hospital Medicines. Trine holds a Master in Public Health Science from the University of Copenhagen and furthermore a Master in Health Services Management from the University of London School of Hygiene and Tropical Medicines / London School of Economics. Trine is especially interested in the generation of Real-World Evidence and has, throughout her career, published +10 peer-reviewed full-scale publications.
Andrzej Rys, MSc
European Commission, Brussels, Belgium
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency, Amsterdam, Netherlands
Dr. Patrice Verpillat is the Head of the Real World Evidence (RWE) Workstream at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. He has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) in order to bring RWE into research, access and life-cycle product management.
Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (efpia) and ISPE.
Petra Wilson, PhD
Health Connect Partners, FTI Consulting, Brussels, Belgium
Petra Wilson is co-founder and managing director of Health Connect Partners, a boutique consultancy which focusses primarily on helping clients understand the European health policy environment. In addition, Petra is acts as EU Policy Advisor to HIMSS and FTI Consulting and is a member of the team at the Institute for Innovation through Health Data, focusing on data governance. Alongside these roles Petra serves on the WHO’s Digital Health Technical Advisory Group; is a Board Member of the European Association for the Study of Diabetes (EASD), and serves on the Ethics Boards of several EU funded projects. Petra holds a Doctorate in Public Health Law from Oxford University, she has British and Belgian nationality and and works in English, German and French.
Petra’s professional experience includes eight years in the European Commission, where she focussed particularly on the use of information society technologies in healthcare; seven years as Senior Director of Connected Health at Cisco, where Petra’s team supported clients in making best use of new communications technologies to drive safer and more efficient access to healthcare. Petra also has deep experience of the health services sector, having worked on both the patient and provider side as CEO of the International Diabetes Federation; as well as experience in academia having been member of the teaching staff of both the law school and medical school of Nottingham University
9:30 - 19:00
Exhibit Hall Open
9:45 - 10:15
Coffee Break
Located in the Exhibit Hall
10:15 - 11:15
Case Studies
RWE Case Studies
These case studies involve the use of real-world data (RWD) and evidence to improve healthcare practices, decision-making, and patient outcomes. The studies address challenges in increasing the uptake of biosimilar medicines, collecting patient-reported outcomes (PROs), evaluating the quality of RWD in economic evaluations, and utilizing RWD to assess the effectiveness of treatments during the COVID-19 pandemic. The stakeholders involved are healthcare organizations, government agencies, HTA agencies, and industry, with an emphasis on collaboration and evidence-based decision-making.
Moderator
Shahid Hanif, PhD, MSc
GetReal Institute, Utrecht, Netherlands
Shahid Hanif is the Managing Director of the GetReal Institute, a non-profit member-led multi-stakeholder association based in the Netherlands, which aims to facilitate the adoption and implementation of real-world evidence in regulatory, HTA and clinical decision-making in Europe. The GetReal Institute has emerged from two Innovative Medicines Initiative (IMI) funded programmes, to establish it as a leading independent and sustainable European forum for stakeholder dialogue, consensus development and co-creation of solutions to advance the use of real-world evidence.
Shahid’s work over the past 20 years includes a breadth of experience in projects, policy and advocacy, relating to health data, digital and outcomes in the life sciences working with pharmaceutical companies and trade associations, public bodies, academic, non-profit and patient organisations.
He co-founded an International Forum for Science Policy to bring together a diverse group for thoughtful deliberation of key issues as it relates to accelerating innovation for the benefit of patients. He is an Associate of RAND Europe and an Affiliate of the Usher Institute at the University of Edinburgh. Shahid holds Bachelor’s and Doctorate degrees in Biochemistry and Molecular Biology, and a Master’s degree in Information Technology from the University of Glasgow.
CS3: Do Real World Data Used to Populate Economic Evaluation of Health Technologies Meet the Requirements of HTA Agencies and Payers: Lessons From a French National Authority for Health (HAS) Case Study (2016-2022)?
10:30AM - 10:45AM
Ghabri S 1 , Sion M2 1 French National Authority for Health (HAS), Saint-Denis La Plaine, France, 2 French National Authority for Health (HAS), Saint-Denis La Plaine, 75, France
Problem Statement : This case study
assessed the quality of real-world data (RWD) incorporated in economic evaluation (EE) appraisals of innovative health products (IHP) performed by HAS from 2016 to 2022, as well as identified limitations related to their use.
Description : The EE appraisals of IHP are performed by HAS for price negotiation between the French Economic Committee of Health Products (CEPS) and pharmaceutical companies. There is a need to evaluate the extent to which these data are adequately used to meet the HAS requirements and the decision makers’ expectations. An in-depth analysis of RWD examples was performed using a RWD reporting guidance and a grid analysis; 138 appraisals of EE identified and assessed the main sources of RWD used to populate the EE (e.g., cohorts used in the external validation of survival outcomes of the standard of care).
Lessons Learned : The use of RWD in EE appraisals of IHP were subject to important limitations and uncertainties (e.g., a lack of comparability of used registries or cohorts describing the disease or the standard of care with the French population of the drug indication, insufficient documentation of the external validation of the long-term effectiveness of the comparator(s)) in almost half of appraisals. EEs were invalidated in 5% of the appraisals due to insufficient Real-World Evidence (RWE) related to the benefit of the studied IHP in terms of effectiveness or quality of life.
Future update of the HAS doctrine may include recommendations improving the assessment of the quality of RWD used in EE (e.g. type of RWD, requirements in terms of specific documentation of the drug adherence and external validation of the long-term survival outcomes and health-state utilities).
Stakeholder perspective : HTA agency
CS4: Informing Clinical Guideline Development With Real-World Data During the COVID-19 Pandemic: Complementing Clinical Trials Real-Time
10:45AM - 11:00AM
Chandak A1 , Mozaffari E2 , Read S 3 , Casciano R4 , Khachatryan A5 , Gottlieb RL6 1 Certara Inc., NEW YORK, NY, USA, 2 Gilead Sciences, Foster City, CA, USA, 3 Certara Inc. Evidence & Access, Edinburgh, Midlothian, UK, 4 Certara Inc., NEW YORK, Poland, 5 Certara Inc. Evidence & Access, London, London, UK, 6 Baylor Scott and White Research Institute, Dallas, TX, USA
Problem Statement : There was an urgent need to identify life-saving therapeutics following the emergence of COVID-19. Findings from clinical trials were limited and evidence from real-world data (RWD) sources offered the opportunity to better characterize the effectiveness and public health value of treatments in specific populations and inform the development of clinical guidelines and support clinical decision-making. However, the rapidly evolving nature of the pandemic posed considerable challenges in the conduct of RWD studies.
Description:
Lessons Learned:
Stakeholder perspective:
CS1: Increasing Biosimilar Uptake With a Novel ‘Best Value-Biological’ Approach in the Irish Healthcare Setting
10:15AM - 10:30AM
Duggan B 1 , Gorry C2 , Browne S2 , Clarke S2 , Barry M2 1 HSE Medicines Management Programme, Dublin 7, Ireland, 2 HSE Medicines Management Programme, Dublin, Dublin, Ireland
Problem Statement: Historically Ireland has had low uptake of generic and biosimilar medicines. The Health Services Executive-Medicines Management Programme (HSE-MMP) was tasked with increasing the prescribing and utilisation of biosimilar medicines for the TNF-α inhibitors adalimumab and etanercept, to 80% market share. TNF-α inhibitors are initiated by hospital prescribers but funded through the community care budget in Ireland. Automatic substitution by pharmacists is precluded by law. A change in prescriber behaviour was therefore required to increase utilisation.
Description: The HSE-MMP published a roadmap for the prescribing of best-value biological (BVB) medicines. This defined criteria to identify preferred biosimilar products for a reference biological medicine. Marketing authorisation holders were encouraged to submit evidence to support their designation as BVB medicines. In addition to identifying a BVB, the HSE-MMP in conjunction with the HSE-Corporate Pharmaceutical Unit and HSE-Primary Care Reimbursement Service:
Developed and implemented a reimbursement policy ensuring exclusive use of BVB medicines in patients initiating treatment.
Implemented a gain-share incentive to support investment of a portion of the savings into the clinical service responsible for those savings, to encourage switching of patients established on originator brands.
Integrated BVB medicine prescribing recommendations into the national reimbursement claims software.
Engaged clinicians and developed support materials for clinical teams.
Lessons Learned: Since June 2019 over 24,500 patients have been prescribed a BVB medicine for adalimumab or etanercept, with associated savings in excess of €80 million. Operation of the gain-share incentive was critical in changing prescriber behaviour, resulting in almost €10 million reinvested in clinical services. Interdepartmental cooperation across the HSE and integration into software systems was critical to success. The BVB programme is now the formally recognised mechanism for biosimilar introduction in the framework agreements between the HSE, Department of Health and industry representative bodies in Ireland.
Stakeholder perspective: Health Services Executive Ireland-Payer/Government Organisation
Breakouts: IP, WS, & OBS
Embracing the AI Revolution: Exploring the Promising Future of Generative AI in HEOR
Digital Conference Pass
Level: Introductory
ISSUE: Should we accelerate the use of generative AI in all domains of HEOR?
OVERVIEW: The panel will delve into the ongoing advancements in generative AI and LLMs within HEOR, highlighting potential benefits, envisioning future trajectory and discussing concrete examples of generative AI producing robust outputs in evidence synthesis and economic modelling.
Tim Reason will introduce the audience to the dynamic field of generative AI, particularly focusing on Large Language Models (LLMs) and their transformative potential in healthcare. He will provide a comprehensive overview of AI and LLMs, defining key terminology, providing historical context, and emphasising how these technologies can enhance patient care, optimise resource allocation, and foster innovation in healthcare. Julia Langham will discuss the current state of generative AI in HEOR, highlighting suitability of LLMs for HEOR-related tasks compared to previous AI approaches. She will showcase concrete examples of how LLMs can generate valuable and accurate outputs in systematic literature reviews (SLR), network meta-analyses (NMA), and economic modelling, underscoring their practical utility in HEOR research. Sven Klijn will present a broader industry perspective on AI and LLMs, exploring key areas such as the potential implications of substantially reduced costs in generating robust scientific materials and insights within the HEOR industry. Additionally, he will discuss the broader concept of technological disruption, drawing insights from "the innovator's dilemma".
Each speaker will present their perspective in a scientifically-grounded manner within a 10-minute timeframe, followed by a 30-minute audience discussion and debate. Attendees, including stakeholders in healthcare, AI researchers, health economists, and policymakers, will gain valuable insights.
NOTE: There will be a discussion group on this topic following the session. Join us in the Discussion Lounge in Hall E North from 11:45-12:45 in Discussion Group A.
Moderators
Bill Malcolm, MSc
Bristol Myers Squibb, Middlesex, LON, United Kingdom
Bill Malcolm leads the Economic & Predictive Modeling group within the Advanced Scientific Capabilities function of global HEOR for Bristol Myers Squibb. Bill has worked across a host of therapeutic areas and roles in pharma over a period of 21 years.
Panelists
Sven Klijn, MSc
Bristol-Myers Squibb, Utrecht, ZH, Netherlands
Sven Klijn is director at Bristol Myers Squibb in the HEOR Economic & Predictive Modeling group, where he leads the innovative modeling agenda in hematology. In addition, Sven has an active role in providing modeling education and masterclasses at international congresses. He has widely published on innovative methods, especially in the field of survival extrapolation. Sven has a training in public health and health economics and previously had various roles in CROs related to health-economic modeling.
Julia Langham, MSc, PhD
London School of Hygiene & Tropical Medicine, London, LON, United Kingdom
Julia Langham is an Epidemiologist, with over 20 years' experience with expertise in the design and analysis of epidemiological studies, systematic review methodology, meta-analysis, and critical appraisal. She has conducted many SLRs in different clinical areas, and multiple prospective and retrospective observational studies both using data collected and existing real-world healthcare databases, leading in the design, analysis and interpretation of studies in both academic and consultancy settings. She teaches on the ClinicalTrials MSc @ LSHTM and she has been involved with the recent Estima project to automate elements of systematic reviews, NMAs and RWE.
Tim Reason, MSc
Estima Scientific Ltd, South Ruislip, LON, United Kingdom
Value Attribution Frameworks for Combination Treatments: Are They a Viable Solution to Solving the Market Access Challenges?
Digital Conference Pass
Level: Intermediate
ISSUE: Patient access remains challenging given the legal challenges inhibiting cross-company dialogue, the difficulty of attributing appropriate proportional value to the individual components of combination therapies, and the monotherapy-dominant paradigm employed in HTA processes and negotiations. To address the value attribution challenge, two value attribution frameworks have been developed (Briggs, et al. 2021; OHE, 2022). These frameworks attempt to define value via formulaic approaches; however, they have been developed for cost-effectiveness markets, particularly with the UK context in mind, where an explicit threshold exists. Further, they only address one component of the issue and require integration within a broader approach encompassing other solutions to address legal and reimbursement challenges. As HTA bodies contemplate the relevance and operationalisation of these frameworks within local methods and processes, careful consideration is warranted to inform their role within a comprehensive set of solutions to a complex challenge. Many questions require consideration: Are they applicable to non-cost-effectiveness markets? Can they be embedded in existing value frameworks and processes? What in addition is required for their implementation?
OVERVIEW: Panel will commence and end with an interactive poll to engage the audience. Speakers from multiple perspectives will be invited to discuss the merits and limitations of two proposed value attribution frameworks, which will be introduced by Jennifer Gaultney (5 minutes). The industry point of view will be presented by Celine Fernandez, representing a manufacturer of emerging oncology combination treatments (12 minutes). Professor Steven Simoens will present a technical critique of the proposed frameworks and their implications in practice (12 minutes). Dr. Dan Ollendorf will present the HTA perspective on the solutions to address the challenges and the role a value framework could have within HTA (12 minutes). A question and answer session will follow each panelist presentation. Panel ends with key take-aways and a concluding polling of the audience.
Moderators
Jennifer Gaultney, PhD, MPH
IQVIA Ltd, London, United Kingdom
Dr. Jennifer Gaultney is an experienced health economist with >10 years of experience in HTA. In her role as Director or health economics and HTA at IQVIA, she leads various engagements supporting clients with developing and implementing their evidence generation plans for HTA purposes. She regularly publishes in health economics and policy journals. She co-founded IQVIA's HTA Centre of Excellence.
Panelists
Céline Fernandez, MBA
Sanofi, GENTILLY, France
Céline Fernandez is the Global Market Access lead for the onco-hematology portfolio and market access policies at Sanofi. She has 15 years of experience in market access and pricing at the global and national level. Before joining Sanofi, she was head of the market access team in the French affiliate at Boehringer Ingelheim coming from Simon-Kucher & Partners, a global pricing and market access consultant agency. Céline earned her doctorate in veterinary medicine from the Lyon University (France) and her master’s degree in business administration from Leonard de Vinci University in Paris, France.
Daniel Ollendorf, PhD
Tufts Medical Center, Boston, MA, USA
Dan Ollendorf is Director of Value Measurement and Global Health Initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR). His research interests include expanding the use of health technology assessment in low- and middle-income economies, as well as refinement of value assessment tools in the US. Prior to joining CEVR, Dr. Ollendorf was Chief Scientific Officer for the Institute for Clinical and Economic Review (ICER) for over 10 years, where he was responsible for scientific oversight of all clinical and economic aspects of the health technology assessment process. As of November 2023, Dr. Ollendorf has returned to ICER as Chief Scientific Officer and Director of International HTA Methods and Engagement.
Dr. Ollendorf holds a PhD in clinical epidemiology from the University of Amsterdam and an MPH from Boston University. He is the current Chair of the Health Technology Assessment International Global Policy Forum as well as a non-resident Fellow at the Center for Global Development, and served on the US Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) from 2015-2019. Dr. Ollendorf has authored over 125 articles in peer-review journals and is co-author of The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press).
Steven Simoens, PhD
KU Leuven, Leuven, VBR, Belgium
Steven is a senior health economist with expertise in economic evaluation, budget impact analysis, market access policy and incentives. He has a particular interest in oncology drugs, orphan drugs, biosimilars and generics, vaccines, antibiotics, cell and gene therapies.
The Role of Patient Preferences in Economic Evaluations: Barriers and Opportunities for a Patient-Based QALY
Digital Conference Pass
Level: Introductory
ISSUE: Practically everyone agrees that patient engagement is an essential component of health technology assessment (HTA): Obviously, the patient's perspective needs to be at the centre of health policy decision making! A patient-based QALY – i.e. QALYs that reflect the preferences of patients, rather than members of the general public – could provide valuable insight and enrich the evidence base for HTA, by quantifying the value of new technologies from the patient’s perspective. However, there are considerable uncertainties around what methods to use to elicit preferences, and how to incorporate them into economic evaluations and decision making. The lack of guidance on these issues may partly explain the significant gap between the theoretical potential of patient preference data and its real-world implementation.
In this session, we will discuss the potential of a patient-based QALY, and the challenges and opportunities associated with the use of patient preference data. The panellists, together with the audience, will explore what steps need to be taken to ensure that patient preferences are incorporated into HTA in an effective and meaningful way. The panel will cover diverse perspectives, including academic, HTA body, and industry. OVERVIEW: This session will explore the role of patient preferences in economic evaluations and policy decision making. The moderator, Dr Paul Schneider, will introduce the topic and outline the historical context of patient-based QALYs. Then each speaker will have 10 minutes to present their views on the issue, followed by a 20-minute audience discussion and debate.
The session will be of interest to a wide range of stakeholders including academics, patient organisations, and decision makers. It will provide a platform for an informed debate on the potential of patient preference data and the challenges and opportunities associated with its practical implementation in economic evaluations.
Moderators
Paul Schneider, PhD, MD, MSc
Valorem Health, Bochum, NW, Germany
Paul Schneider is a postdoc at the University of Sheffield and the founder of Valorem Health. He works on methodological and normative issues in the valuation of health and preference elicitation.
Panelists
Martin Henriksson, PhD
Linköping University, Linköping, Sweden
Martin Henriksson is an Associate Professor of Health Economics at Linköping University. His research is primarily focused on health economic evaluation, but also includes the estimation of experience-based value sets for EQ-5D and health policy issues concerning the interpretation of cost-effectiveness. As a member of the Swedish Pharmaceutical benefits board, he has long experience as a national decision-maker responsible for reimbursement decisions.
Anja Schwalm, Dr.
Institute for Quality and Efficiency in Health Care (IQWiG), Cologne, Germany
Anja Schwalm is one of the heads of the Health Economics Division at the Institute for Quality and Efficiency in Health Care (IQWiG). She is involved in the early benefit assessments of pharmaceuticals with the focus on economic aspects (assessment of the information on the number of patients and the treatment cost provided in the dossier submitted by the drug manufacturer). She is also responsible for the development of IQWiGs methods for health economic evaluations.
Ben A Van Hout, PhD
University of Sheffield, Sheffield, United Kingdom
Ben van Hout combines a position of professor of Health Economics with that of Chief Scientific Officer in OPEN Health Evidence & Access. In 2020, he received the Donabedian Lifetime Achievement award for his contributions to the field of economic evaluations in health care. Over the last couple of years, he has taken his research to the next level by first having a myocardial infarction and second having a large motorcycle accident. Since then, he has taken more than 16,000 tablets, still counting, for prevention, pain and itch.
His current position allows him to only be involved in projects he fancies, “personal utility functions” being on the top of the list.
Issues Regarding Pricing and Access to Gene and Advanced Therapy Medicinal Products
Digital Conference Pass
Level: Experienced
ISSUE: Most payers worldwide are grappling with the challenge of paying for very expensive but effective and potentially curative gene and other advanced therapy medicinal products (ATMPs). An exponential growth in the availability of new gene therapies and ATMPS is expected in the coming decade. Finding solutions on how societies can finance expensive but effective, potentially curative medicinal products in ways that enhance welfare is pressing. There are fundamental disagreements among stakeholders on these issues. In this Issues Panel, panelists will debate whether we can find common ground?
OVERVIEW: Dr. Campbell will moderate a session with Panelists discussing the issues and solutions to this problem. Dr. Steuten will provide an overview of recent and recommended changes to HTA methodologies and evidence-generation activities in nine European countries, plus Canada and Australia, that would enable the full value of ATMPs and gene therapies to be captured in HTA. She will illustrate these activities using examples of best practices internationally. Dr. Briggs will consider the evaluation of ATMPs within a fixed-threshold system such as the UK, including issues of budget impact and possible risk-sharing arrangements. Dr. Basu will discuss the role of budget impact in assessing value-based prices for gene therapies, present a case analysis of pricing gene therapy for sickle cell disease in the US, and demonstrate what different US state Medicaid programs can do to absorb the initial budget impact.
Moderators
Jon Campbell, PhD
National Pharmaceutical Council, Washington, DC, USA
Jon Campbell is Chief Science Officer at the National Pharmaceutical Council (NPC). Jon is a leader in US value assessment. He is an author of over 250 manuscripts and abstracts in the field of value assessment and holds an adjunct faculty position within the Center for Evaluation of Value and Risk in Health at Tufts University School of Medicine. Jon has graduate training degrees in pharmaceutical outcomes research (PhD) and biostatistics (MS) from the University of Washington.
Panelists
Anirban Basu, PhD, MS
University of Washington, Seattle, WA, USA
Anirban Basu is a Professor of Health Economics and the Stergachis Family Endowed Director of The CHOICE Institute at the University of Washington, Seattle. He holds joint appointments with the Departments of Health Systems & Population Health and Economics at UW, is a Faculty Research Fellow at the US National Bureau of Economic Research, and an elected Fellow of the American Statistical Association. His work sits at the intersection of microeconomics, statistics, and health policy. His research focuses on understanding the economic value of health care, generating causal evidence, and, lately, on the potential for discrimination with machine learning and artificial intelligence algorithms. He served on the Second Panel on Cost-effectiveness Analysis in Health and Medicine and serves on the Editorial Advisory Board for Value in Health Journal. He received his master's in Biostatistics from UNC-Chapel Hill and a PhD in Public Policy Studies from the University of Chicago.
Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, LON, United Kingdom
Andrew Briggs is Professor of Health Economics at the London School of Hygiene & Tropical Medicine, and co-Director of the Global Health Economics at the School. He has authored over 300 peer reviewed publications and is a Clarivate 'highly-cited' researcher. He has co-authored two textbooks, one published by OUP entitled Decision Modelling for Health Economic Evaluation, and the other published by Wiley entitled Statistical Methods for Cost-Effectiveness Analysis.
A link to his GooGle Scholar page: https://bit.ly/GS-briggs
Lotte Steuten, PhD
Office of Health Economics, London, Greater London county, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
Spotlight Session
EU Joint Clinical Assessment – One for All and All for One?
Digital Conference Pass
Level: Intermediate
ISSUE:
EUnetHTA-21’s scoping guideline suggests most new medicines undergoing JCA would have a small number of PICOs. However, analysis in a common oncology indication indicates that the stated method could initially lead to >10 PICOs. Furthermore, the unique needs of a MS for outcomes across all PICOs could lead to more analyses than currently requested across Europe.
The PICOs will determine the size of the evidence to be submitted, assessed and reported. The EU HTA Regulation lays out a tight timeframe to deliver this. It may therefore require prioritization and consolidation in the PICOs, as well as sufficient resources, to ensure that a high quality assessment can be undertaken within the timeframe, without delaying time to patient access decisions at Member State level.
OVERVIEW: (10 min) Anke van Engen will open the discussion on how to ensure a transparent, evidence-based approach to producing common European PICOs. Using a case study she will illustrate how implementation of the scoping process as outlined by EUnetHTA21 could be challenging for both industry and assessors leading to a high number of PICOs, many requiring ITC, and substantial analysis requests.
(10 min) Anne Willemsen will respond based on a survey conducted among MS and will suggest how to bridge the gap between industry and HTA bodies in terms of scoping expectations.
(10 min) James Ryan will outline an industry perspective on the proposed process, methodology and implications for evidence development. He will suggest how comparators and populations can be selected in a predictable, consistent, and transparent manner, to deliver on the original vision of the Regulation.
(10 min) Kim Helleberg Madsen will comment on differences between JCA and local scope and what can be expected in terms of additional analysis requests.
(20 min) Facilitated discussion with the audience with the opportunity to ask questions and propose solutions.
Signal—ISPOR’s signature program—looks beyond today’s linear thinking to explore topics that will shape healthcare decision making over the next decade. Seeking to strengthen strategic foresight and adaptive leadership capacities in the complex world of healthcare, the Signal series focuses on the “big picture,” while also addressing how health economics and outcomes research (HEOR) can best contribute to solving healthcare’s greatest challenges.
NOTE: There will be a discussion group on this topic following the session. Join us in the Discussion Lounge in Hall E North from 11:45 - 12:45 in Discussion Group B.
Moderators
Anke van Engen, MSc
IQVIA, Amsterdam, NH, Netherlands
Anke is a Managing Principal and leads the HE/HTA Center of Excellence in the European Value & Payer Evidence practice. She has over 20 years of experience and has co-authored many articles and spoken at international conferences.
Panelists
Kim Helleberg Madsen, MSc
Danish Medicines Agency, Copenhagen, N/A, Denmark
Kim is Director of Pharmacoeconomics & Availability at the Danish Medicines Agency, and a member of the EU HTA CG and chair of the HTA subgroup on Emerging Health Technologies.
James Ryan, MSc
AstraZeneca, Cambridge, CAM, United Kingdom
James represents AstraZeneca on regional and global HTA Policy around the world and is a co-lead on EFPIA’s HTA Working Group’s methodology workstream on the EU HTA Regulation. With over 25 years of experience in health economics and payer evidence generation, James has extensive knowledge and experience of making HTA submissions across Europe and beyond. In addition to his policy role, he also leads the oncology global HTA and Modelling Science team at AstraZeneca.
Anne Willemsen, MSc
Zorginstituut Nederland (ZIN), Diemen, Netherlands
Anne Willemsen works for the Dutch National Healthcare Institute (Zorginstituut Nederland) and has been coordinating Joint Clinical Assessments (JCA) under EUnetHTA Joint Action 3 and EUnetHTA 21. In April 2023, she was elected as the co-chair of the HTA Coordination Group JCA subgroup under the HTA Regulation.
Breakouts: IP, WS, & OBS
Estimands in Clinical Trials and HEOR: Applications and Considerations
Digital Conference Pass
Level: Intermediate
PURPOSE: This workshop will focus on the application of the estimand framework to different analyses in trials. Workshop participants will become familiar with using and interpreting estimands to address different post-randomisation events in trials, including treatment switching and attrition due to mortality or disease progression. Participants will also learn about how commonly used statistical methods in trials, such as time-to-event models, can be applied within the estimand framework.
DESCRIPTION: The ICH addendum on estimands in clinical trials was introduced in 2019 and has since been implemented by regulatory agencies around the world. The goal of this estimand framework is to better link trial design, objectives and analysis. Using estimands can improve communication across trial stakeholders and facilitate clearer interpretation of treatment effects. Applying and interpreting estimands is essential for health economics and outcomes researchers working with trials or trial data. This workshop will cover the application, dissemination and use of estimands for statistical and health economic analyses.
Dr. Roydhouse will open the session with a discussion of the estimand framework and examples of estimand use in practice (10m). Dr Floden will discuss the development of estimands for time-to-event endpoints in oncology settings, where attrition due to mortality, progression and toxicity are frequent (10m). Prof Latimer will discuss handling treatment changes when estimating long-term survival for health economic analyses (10m). Dr Regnault will discuss the analysis of patient-reported outcome data with estimands (10m). The workshop will use real-time polling and discussion with the audience to engage participants and work through questions about the application and use of estimands. The workshop will be beneficial for health economics and outcomes researchers and industry stakeholders working with clinical trials or trial data.
Discussion Leaders
Jessica Roydhouse, PhD
Menzies Institute for Medical Research, Hobart, TAS, Australia
Dr Jessica Roydhouse is Select Foundation Senior Research Fellow in Health Services Research at the Menzies Institute for Medical Research, University of Tasmania, Australia. In this role she also serves as Director of the Tasmanian Cancer Registry. Her research interests include patient-reported outcomes, cancer clinical trials and missing data.
Discussants
Libby Floden, PhD, MPH
Clinical Outcomes Solutions, Tucson, AZ, USA
Libby is a Senior Director of Biostatistics on the Quantitative Team at Clinical Outcomes Solutions, a global Health Economics and Outcomes Research strategic consulting group. Libby is a biostatistician with over 10 years of experience in patient-reported outcomes (PRO) research and clinical trials as an academic researcher and industry consultant. Much of her work has been in the development, interpretation, and analysis of PROs with missing data, specifically when using responder analyses. She specializes in complex quantitative approaches for optimal interpretation, the estimand framework for PROs and has extensive experience in psychometric analyses and mixed-methods research.
Nicholas Latimer, MSc, PhD
The University of Sheffield, Sheffield, DBY, Great Britain
Nick is a Professor of Health Economics at the University of Sheffield, a Yorkshire Cancer Research Senior Fellow, and an Analyst for Delta Hat Ltd. He was a member of NICE Technology Appraisal Committee B for 5 years and is a member of the NICE Decision Support Unit (DSU). Nick’s research focuses on survival analysis in economic evaluations. He has authored four DSU technical support documents related to survival analysis, treatment switching, and partitioned survival models. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of cancer treatments used in clinical practice.
Antoine Regnault, PhD
Modus Outcomes, a division of THREAD, Lyon, 69, France
Antoine is a biostatistician with a strong interest in measurement science applied to patient-centered outcomes. As a consultant with 20+ years of experience, he uses his combined skillset in psychometrics and statistics to support pharmaceutical companies with their analyses of patient-centered outcomes data from clinical trials and observational studies in a wide variety of disease areas (oncology, hematology, neurology and rare diseases, among others). Antoine has also been an active member of several large international patient-centered outcome research initiatives such as SISAQOL-IMI, SPIRIT-PRO or NeuroMET-2.
Using Federated Data Networks and Common Data Models for HTA and Regulatory Evidence Generation: Challenges, Recommendations, and Learnings From IMI, EHDEN, and Darwin EU
Digital Conference Pass
Level: Intermediate
PURPOSE: This workshop aims to explore how regulators and health technology assessment (HTA) agencies can use federated data networks and common data models to generate real-world evidence (RWE) to meet their needs and assess evidence generated using these approaches. It will also explore the opportunities and challenges of using federated data networks to generate RWE.
DESCRIPTION: Federated data networks are collaborative infrastructures that use common data models to facilitate using standardised analytics to generate RWE, while preserving data privacy and security. The Innovative Medicines Initiative (IMI) European Health Data and Evidence Network (EHDEN) and Data Analysis and Real-World Interrogation Network (DARWIN EU) are examples of initiatives that utilise federated networks, aiming to harmonise and standardise real-world data (RWD) to increase interoperability and generate reliable, representative, and actionable RWE. However, federated networks pose their own unique challenges.
In this workshop, Prof Dawoud will introduce the topic of federated data networks and their use to generate evidence (5 minutes). Prof Rijnbeek will present the concept of federated data networks and describe their use in EHDEN, giving examples of use cases (10 minutes). Dr Abellán, will discuss the regulatory perspective on using federated data networks for RWE generation, focusing on DARWIN EU and its use in regulatory and use cases (10 minutes). Prof Goettsch will talk about the recommendations for the incorporation of federated data networks in RWE frameworks and the key learning from their use in use cases from an HTA and payer perspective (10 minutes). The workshop will allocate 20 minutes for an interactive audience discussion, including question and answer sessions. Audience polling will be employed to encourage active participation and engagement.
Discussion Leaders
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Associate Director (Research) at the National Institute for Health and Care Excellence (NICE). She has practised as health economist and researcher for over 20 years. Her current work is focused on advancing HTA and clinical guideline development methods through research.
Dalia leads NICE newly established HTA Innovation Laboratory (HTA Lab) as well as NICE’s portfolio of European Commission funded projects, such as IMI EHDEN and HORIZON 2020 HTx projects, with cumulative funding of over 5 Million Euros. She is widely published in the area of health economics and outcomes research and serves as Associate Editor of ISPOR journal Value in Health and as Director on ISPOR Board of Directors (2023-2026).
Discussants
Juan Jose Abellan, PhD
European Medicines Agency, Amsterdam, Netherlands
Juan Jose Abellan is a mathematician and statistician by training. He has worked as a statistician in various roles in Public Offices, Academia and the Pharmaceutical Industry in Spain, Germany and the UK. He has worked in several scientific disciplines such as Epidemiology, Metagenomics and Clinical Drug Development, and he has made a number of contributions in those fields in peer-review scientific journals. He is currently working for the European Medicines Agency (EMA) based in the Netherlands and is part of the EMA team developing the Data Analysis and Real World Interrogation Network (DARWIN EU) for the generation of real-world evidence to support regulatory decision making around the utilisation, effectiveness and safety of medicines.
Wim Goettsch, PhD
National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He has serves as a special HTA advisor to Zorginstituut Nederland and a former director of EUnetHTA JA3 Directorate.
Peter Rijnbeek, PhD
Erasmus MC, Rotterdam, Netherlands
Peter Rijnbeek is Professor of Medical Informatics and is the Chair of the Department of Medical Informatics of the Erasmus MC, The Netherlands.
He leads the research line Health Data Science (HDS, www.healthdatascience.nl). He obtained his MSc (1996) in Electrical Engineering at the Technical University Delft. His PhD thesis, received from the Erasmus University Rotterdam, was on the development of a computer program to automatically interpret pediatric electrocardiograms.
The HDS team has a leading role in the Observational Health Data Sciences and Informatics (OHDSI, www.ohdsi.org) by co-leading the Patient-Level Prediction workgroup and contributing to methods and tool development in the OHDSI eco-system. His team addresses issues that deal with the quality of data, the translation of data to other formats, mechanisms to access data in a federated fashion, and the development of analytical pipelines for characterization, population-level effect estimation, and patient-level prediction. Prof. Rijnbeek is lead of the European OHDSI Chapter (www.ohdsi-europe.org) that promotes and enables the adoption of the OMOP-CDM in Europe. He is the coordinator of the European Health Data and Evidence Network (EHDEN) IMI project that builds a federated network of health data bases across Europe standardized to the OMOP (www.ehden.eu). Furthermore, Prof Rijnbeek is the executive director of the DARWIN EU® Coordination Centre that manages a network of real-world healthcare data sources across the EU to conduct scientific studies requested by the European Medicines Agency (EMA).
More information and a publication list can be found here: https://www.healthdatascience.nl/peter-rijnbeek-cv
Podium Sessions
Novel Approaches to Patient Communication, Data Collection, and Involvement in Research
Digital Conference Pass
Moderator
Irina Kinchin, PhD, MSc
Trinity College Dublin, the University of Dublin, Dublin, D, Ireland
P41: Assessing the Role of Patient Feedback in the National Institute for Health and Care Excellence (NICE) Decision Making in the UK: Are Patient Voices Truly Heard?
10:15AM - 10:30AM
Pandit U
OBJECTIVES: In 2019, the National Institute for Health and Care Excellence (NICE) found that patient organisations perceived their current involvement as insufficient, and that more transparency was needed when accounting for patient feedback in the final decisions. We aim to assess how the role of patient input is presented in NICE decision making since 2019.
METHODS: All NICE Final Appraisal Determinations (FADs) – describing the decision-making – published between January 2021 – January 2023 were reviewed. Terminated appraisals were excluded due to insufficient documentation. Using a pre-determined extraction sheet the main characteristics of the appraisal (e.g., type, disease area, rarity, patient population, and final recommendation), areas of patient input (e.g. prognosis, unmet need, clinical outcomes, treatment pathway, equality considerations, patients quality-of-life (QoL), impact on family/caregivers, impact of side effects) and details of the input (e.g. quantitative/qualitative) were recorded. Results were analysed using summary statistics and compared to previous results from the literature.
RESULTS: 201 FADs were screened of which 43 were excluded. The majority of appraisals were in chronic diseases (93%) with 51% of all FADs in oncology. 19% of FADs were in rare diseases and the majority of technologies (92%) received positive recommendation. Patient input was mentioned in 87% of FADs. Input was most frequently reported for QoL (60%), unmet need (43%) and clinical outcomes (42%). 4% of appraisals used quantitative input from patients. While all the rare disease FADs recorded patient input, none were quantitative. There was no clear pattern between NICE recommendations or disease area or chronic nature and patient input. Compared to previous reviews, the extent of patient input has increased.
CONCLUSIONS: NICE has made important progress in including patient input in the decision-making process; however, the range of areas is limited. There is also further opportunity to explore quantitative patient input, especially in rare diseases where data is limited.
P44: Seeing it in the Flesch: Comparing Readability Between AI-Generated and Human-Written Plain-Language Abstracts
10:45AM - 11:00AM
McMinn D 1 , Valena T2 , Bender W3 1 Sorcero, East Kilbride, SLK, UK, 2 Sorcero, Basel, NA, Switzerland, 3 Sorcero, Washington, DC, USA
OBJECTIVES: Plain language summaries of scientific content are emerging as a key area for the application of AI in the field of patient communications. This study compares readability scores of AI-generated plain-language abstracts with those written by humans.
METHODS: A total of 48 human-written (HW) plain-language abstracts and their corresponding scientific (SC) abstracts were obtained via a PubMed search. An AI-assisted plain-language abstract (AI) was auto-generated for each of the 48 SC abstracts by using a combination of programming instructions and several large language models—including BioBERT and the Pathways Language Model (PaLM). Readability scores were calculated for all HW, SC, and AI abstracts using Flesch-Kincaid, Automated Readability Index (ARI), Linsear-Write, and SPACHE. Readability scores were compared between abstract types using ANOVA with Bonferroni pairwise comparisons. A subject-matter expert quality analysis was conducted on AI abstracts using a scale of Good, Moderate, and Poor. Analyses were conducted before and after AI model enhancement and the introduction of content validation steps.
RESULTS: Before model enhancement and content validation, mean Flesch-Kincaid, ARI, Linsear-Write, and SPACHE age/grade-level scores were 16.5, 17.7, 20.4, and 9.9 for SC abstracts, 12.3, 12.8, 13.7, and 7.6 for HW abstracts, and 8.7, 8.5, 8.2, and 6.2 for AI abstracts. Age/grade-level scores were significantly lower (i.e. easier to read) for AI vs. HW abstracts for all readability metrics (p<0.01). Following model enhancement and content validation, AI abstract readability scores were 8.5, 8.4, 8.3, and 6.0. Furthermore, the percentage of AI abstracts categorized as ‘Poor’ quality decreased from 22% to 12% and the percentage categorized as ‘Good’ increased from 33% to 45%.
CONCLUSIONS: AI-assisted auto-generated plain-language abstracts of scientific content are easier to read than those written by humans. Further studies will be conducted to investigate labor-saving and quality-enhancing benefits of these technologies for the development of plain language materials.
P42: Enhancing the Understanding of Phenotypic Outcomes in Sickle Cell Disease (SCD) Through a Comparison Between Data Collected Through App-Based Home-Reported Outcomes (HROs) Versus Qualitative Methodologies
10:30AM - 10:45AM
Zhang C1 , Healey A 2 , Anwar H2 1 Folia Health, Kansas City, MO, USA, 2 Folia Health, Boston, MA, USA
OBJECTIVES: To compare a novel app-based data capture framework with qualitative data collection in the sickle cell disease (SCD) population.
METHODS: A literature search identified unique concepts and themes in qualitative data previously collected in SCD. These concepts were compared against an outcome inventory created from a group of SCD participants using the app-based Folia home-reported outcomes (HROs) platform. The outcome inventory was further categorized using patient-led concept grouping and mapped to relevant domains based on clinical judgment and subject matter expertise.
RESULTS: Qualitative data collection methods, such as interviews, diaries, and surveys using patient-reported outcome (PRO) measures, provide insight into the subjective burden of SCD. In contrast, direct app-based tracking allows participants to identify and track their experiences flexibly and at an individual level. Aggregated over a period of time, this app-based data collection method creates an outcome inventory - a granular dataset of symptomatology for a specific condition-of-interest.
A comparative analysis between the outcome inventory captured in Folia and themes from qualitative SCD data will be presented. Semi-structured interviews, patient-reported diaries, and PRO surveys produced a variety of concepts ranging from 8 domains to 30 domains.The Folia outcome inventory applied to tracking data of 82 SCD participants resulted in 92 unique symptoms across 8 categories. Compared to qualitative data capture, the outcome inventory revealed over 20 different manifestations of pain, and 12 types of behavioral/mood symptoms. This outcome inventory methodology captured similar concepts to semi-structured interviews but with higher volume and greater detail, including real-time symptom experiences and healthcare-seeking behavior. CONCLUSIONS: In designing a study to understand the nuances of SCD, individualized and participant-driven research methods - such as utilizing an app-based outcome inventory - may enhance existing knowledge. The outcome inventory methodology provides a detailed understanding of SCD burden, contributing to a comprehensive assessment of the disease.
The Impact of Cost-Containment Initiatives From Across the Globe
Digital Conference Pass
Moderator
Timm Volmer, Dr.
SmartStep Data Institute, Hamburg, Germany
P8: The Dutch Lock Procedure for Reimbursement Works as Intended
10:45AM - 11:00AM
Boer J 1 , van Straaten W2 , Tjon Joe Gin L2 , Boonen L2 1 Equalis Strategy & Modeling, Utrecht, UT, Netherlands, 2 Equalis Strategy & Modeling, Utrecht, Utrecht, Netherlands
OBJECTIVES: The Dutch government introduced a lock procedure in 2015 to regulate access of expensive hospital drugs to the market. The lock intents to prevent uptake of ineffective drugs and to negotiate the price of effective drugs to regulate the impact on the health care budget.
METHODS: Using technology assessment reports from the Dutch Health Care Institute, we developed a method to quantify the effects of the lock. We used QALYs to quantify the health effect and also created insight in the budget impact of the price negotiations. The effects are calculated for the time during and after the lock, for which we used different scenarios. We estimated a total effect retrospectively, based on all drugs that completed the lock procedure between 2015 and 2021.
RESULTS: A total of 29 drugs have been included. The results indicate that the total health effect of drugs placed in the lock between 2015 and 2021 is a population health gain of 5,551 to 10,772 QALYs, dependent on the comparative route, using a time horizon of 5 years. The budget effect is a saving between €906 million and €1,185 million.
CONCLUSIONS: The results show that the lock does not capitalize on the potential health benefits of new innovative drugs in the short term, but that it limits health loss due to displacement in the long term. The long-term effects outweigh the short-term exclusion of new drugs from the market. These effects depend not only on the potential health benefits of the new drug, but also on the lead time of the comparative route and the price being negotiated. Policymakers can use these results to quantify the impact of their reimbursement procedures and stress the importance of a lock procedure.
P7: International Reference Pricing and Joint Clinical Assessments—How Will European-Level HTA Evaluations Impact International Reference Pricing and Launch Sequencing?
10:30AM - 10:45AM
Saei A1 , Tutulea E2 , Perez-Kempner L 3 1 Parexel International, Stockholm, Stockholm, Sweden, 2 Parexel International, Bucharest, B, Romania, 3 Parexel International, Lebrija, SE, Spain
OBJECTIVES: While international reference pricing (IRP) has traditionally been used to inform launch sequencing, European efforts to standardize the determination of clinical value for new products through joint clinical assessments (JCAs) are expected to impact launch preparedness. This research aims to analyze launch sequences of drugs to evaluate if launch preparedness is still heavily driven by IRP policies or is incorporating components to account for the future effects of JCAs.
METHODS: We selected 13 countries with established HTA entities that have IRP policies or act as IRP referenced countries and 10 multiple sclerosis (MS) drugs as the crowded MS spaced allowed for a robust drug sample despite the challenges of a mature market. We defined the launch sequences based on HTA submission dates, considering the average HTA process duration and excluding submissions preceding 2019. Finally, we analyzed launch sequences and IRPs to determine whether the latter may have informed the launch preparedness.
RESULTS: Overall, 5/10 drugs had six or more HTA submission dates that informed the launch sequence. The first-launch country was UK (n=3/5), Australia, or Norway (n=1/5 each). UK was most frequently followed by Norway (n=2/3), followed by Australia (n=1/3). Sweden was most often positioned as the fourth and Germany as the fifth/sixth country. France positioning ranged from second to seventh (n=1 each). Romania, Denmark, Poland, and Brazil were positioned after their IRP referencing countries. Launch sequences were consistent with IRP policies for 4/5 drugs, where companies launched first in non-IRP-guided countries (Australia) or countries negotiating confidential discounts (UK).
CONCLUSIONS: Manufacturers are still relying on IRP policies to inform their launch sequences. As JCAs policies start to be implemented, the influence of IRP on pricing decision-making is expected to decrease. Global pricing research and early engagement with JCAs stakeholders will likely become critical to inform new launch preparedness plans in the upcoming years.
P5: An Update on Calcitonin Gene-Related Peptide Monoclonal Antibodies Reimbursed Under a Managed Access Protocol in Ireland
10:15AM - 10:30AM
Finnigan K 1 , Smith A2 , Clarke S3 , Barry M3 , Gorry C3 1 HSE Medicines Management Programme, Dublin, Ireland, 2 HSE Medicines Management Programme, Dublin 8, Ireland, 3 HSE Medicines Management Programme, Dublin, Dublin, Ireland
OBJECTIVES: The calcitonin gene-related peptide (CGRP) monoclonal antibodies (MABs) erenumab, fremanezumab and galcanezumab are reimbursed in Ireland under the High Tech Arrangement for the prophylaxis of chronic migraine in adults who have failed three or more prophylactic treatments. Reimbursement is subject to a Health Service Executive (HSE)-Managed Access Protocol (MAP), introduced on 1st September 2021. This study provides a summary of the number of applications for CGRP MABs submitted and an overview of the utilisation of CGRP MABs on the High Tech Arrangement.
METHODS: All reimbursement applications for CGRP MABs submitted to the HSE-Medicines Management Programme (MMP) through an online application system between 1 September 2021 and 30 April 2023 were reviewed. Data was extracted from the HSE-Primary Care Reimbursement Services (HSE-PCRS) national pharmacy claims database for the High Tech Arrangement, from 1 September 2021 to 30 April 2023. Analysis was performed using SAS® 9.4 Software.
RESULTS: A total of 1,509 reimbursement applications were submitted between September 2021 and April 2023. Reimbursement has been approved for 96.2% (n= 1,452) of applications submitted.
There were 1,399 individual patients dispensed a CGRP MAB under the High Tech Arrangement between September 2021 and April 2023. The majority of patients in receipt of CGRP MABs were female (n= 1,141, 81.6%) and had a mean age of 45 years. In April 2023, 1,069 individual patients were dispensed a CGRP MAB on the High Tech Arrangement. The proportion of patients in receipt of individual CGRP MABs was 56% (n= 599) fremanezumab (Ajovy
® ), 38.3 % (n=409) erenumab (Aimovig
® ), and 5.7 % (n=61) galcanezumab (Emgality
® ).
CONCLUSIONS: When used in conjunction with health technology assessment, MAPs, an example of health technology management, enable access to new drug treatments for patients likely to derive most clinical benefit, while providing budgetary oversight and certainty for the payer.
Case Studies
Member Group Meetings
ISPOR Algeria Chapter Meeting
This meeting welcomes all those with an interested in the ISPOR Algeria chapter. It’s a fantastic opportunity to dive deeper into the chapter’s activities and discover how to actively participate.
10:30 - 13:30
Poster Session 1
Live
11:15 - 13:15
Lunch
11:30 - 12:00
Fast Facts
How to Get Your Papers Published in Peer-Reviewed Journals
Level: Introductory
In this session the Editors-in-Chief of Value in Health will give an 'insiders' view of the article evaluation processes of peer-reviewed journals. In doing so, they will offer some tips of what to do, and not to do, when preparing and submitting your papers.
Discussion Leader
Michael Drummond, PhD
University of York, Lichfield, Staffordshire, United Kingdom
Michael Drummond, BSc, MCom, DPhil is professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 700 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
C. Daniel Mullins, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor at the University of Maryland School of Pharmacy. He is founder and executive director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program. He received his BS in Economics from M.I.T. and his PhD in Economics from Duke University. His research and teaching focus on comparative effectiveness research (CER) and patient-centered outcomes research (PCOR). Dr. Mullins has received funding as a Principal Investigator from AHRQ, FDA, NHLBI, NIA, NIMHD, Patient-Centered Outcomes Research Institute (PCORI) and various patient advocacy organizations and pharmaceutical companies. He is the lead for the Community & Collaboration (C&C) Core of the University of Maryland Institute for Clinical and Translational Research (ICTR) and co-lead of the C&C Core for Johns Hopkins’ CTSA.
Professor Mullins is 1 of 2 editors-in-chief for Value in Health and is author of over 325 peer-reviewed articles and book chapters. At the University of Maryland Baltimore (UMB), he received the Dr. Patricia Sokolove Outstanding Mentor Award and the Dr. Martin Luther King Jr. Faculty Diversity Award. He was named Researcher of the Year at UMB and was awarded a University System of Maryland Wilson H. Elkins Professorship. He is a past recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award, and the ISPOR Marilyn Dix Smith Leadership Award.
: C. Daniel Mullins
11:45AM - 12:00PM
Mullins CD
: Michael Drummond
11:30AM - 11:45AM
Drummond M
11:30 - 12:15
Poster Tours
Real-World Evidence in Europe Poster Tour
Posters featured in this tour:
PT7: Data Standardization in Brazil: An OMOP Common Data Model Approach in a DATASUS Cohort
PT8: Five-Year Healthcare Resource Consumption and Direct Costs of Women with a New Diagnosis of Hr+/HER2- Breast Cancer Primary or Advanced: Analysis of a Large Italian Administrative Database
PT9: Harnessing the Potential of Natural-Language Processing and Interconnected Data Streams for Complex Diseases in the Hospital Setting: Lupus Case Study in France (LUPUS REAL)
PT10: Opioid Tapering and Its Associations with Risk of Opioid Use Disorder and Mortality Among Older Adults
PT11: Overall Survival of Patients with Lung Cancer Newly Diagnosed in the Period of the COVID-19 Pandemic Compared to Patients with an Incident Diagnosis Before the Pandemic: A Retrospective Analysis of German Claims Data
PT12: Perceived Financial Burden and Quality of Life Among Patients with Cancer
Sponsor
Moderator
Louise Hartley, PhD
RTI Health Solutions, Didsbury, Manchester, LAN, United Kingdom
Patient-Centered Poster Tour
Posters featured in this tour:
PT1: A Comparison of the UK and Australian Interim Value Sets for the Weight-Specific Adolescent Instrument for Economic Evaluation (WAItE)
PT2: Comparing UK Value Sets and Mapping Functions for EQ-5D
PT3: Concept Elicitation Interviews to Refine a Conceptual Model of the Patient Experience of Amyotrophic Lateral Sclerosis (ALS)
PT4: Patient-Reported Outcomes (PROs) in German AMNOG-Assessments: Impact of Increasing the Response Threshold to 15 %
PT5: The Role of EUnetHTA 21 in Promoting Patient Engagement
PT6: What Do Patients Value? Development of a Preference-Based Health Utility Score for Chronic Obstructive Pulmonary Disease (COPD)
Sponsor
Moderator
Sam Llewellyn, MPH
Vitaccess, Oxford, OXF, United Kingdom
Sam is Associate Director of Patient Centered Research at Vitaccess.
Sam provides direction and oversight to a range of projects across our health economics and outcomes research, PCO, and real-world evidence portfolio. Sam’s day-to-day responsibilities include study design and implementation, ethical approval strategy and submissions, conduct and analysis of Delphi panels and qualitative interviews, and publication development.
Sam joined Vitaccess in 2018, after spending three years as a project manager within the PCO group at a leading contract research organization, where Sam managed large, multi-country outcomes research studies. Sam has worked across a broad range of therapeutic areas, including gastrointestinal, oncology, respiratory, and rare neurodegenerative diseases.
New Attendee/New Member Welcome Event
Is this your first time attending an ISPOR conference, or are you a new member? Come by the ISPOR Booth to learn how to maximize your conference experience and make the most of ISPOR membership!
11:45 - 12:15
Exhibit Hall Theater
Artificial Intelligence and Machine Learning Tools at the Heart of Future NICE Evaluations: A Prospect or a Pipe Dream?
Digital Conference Pass
Over the last decade we have experienced an exponential growth in both the broad interest in & the volume of publication around the theoretical & ethical considerations of using Artificial Intelligence & Machine Learning methodologies within the health sector. What we are seeing is a clear move towards a phase of developmental adoption. The focus is on expanded practical development, with AI/ML based tools being trialled in the support of the key analytical & evidence gathering process stages within HTA /HEOR programmes.
Evidence screening, gathering & baseline synthesis (aligned with the classic use of SLR-based approaches & descriptive data summary in the HTA process)
Data analytics & hypothesis testing (providing an approach to deal with large & complex clinical data sets to provide short-list suggestions for patient cohorts & clinical effect groups)
Predictive data-driven modelling of outcomes
As this pace of development continues, what remains unclear is how can we ensure that, as HTA practitioners & Payer decision makers, we retain common standards of applied rigour & expectations with respect the use of AI/ML in supporting HTA evaluation & decision making.
Our expert panel members will provide their own unique practical perspectives & thoughts around three specific questions:
Where do you feel we’ll begin to see the first wave of formal inclusion in HTA method guidelines, in terms of the practical applications for AI/ML alongside established HTA / HEOR methodologies?
What do you see as the primary practical advantages in opening our existing processes to AI/ML enhancement within the context of HTA / HEOR?
What do you feel are the necessary steps from HTA bodies (such as NICE) to ensure the move to include AI/ML is done in a quality-assured, consistent, ethical, and transparent way?
Sponsored by AXIS Healthcare Consulting Ltd
Sponsor
AXIS Healthcare Consulting Ltd
Moderators
Brenda Dooley
AXIS Healthcare Consulting Ltd, Dublin, D, Ireland
Brenda Dooley is the Founder and CEO of AXIS Consulting, a leading provider of strategic market access and health technology assessments for pharmaceutical and biotechnology firms. Working predominantly within the Irish and UK markets, the company assists businesses across the pharma industry to successfully navigate complex healthcare environments.
An experienced market access strategist with more than 20 years' international biopharma industry experience, Brenda established the business in 2012, and has developed over 120 bespoke and high-quality reimbursement submissions for the Irish authorities. Brenda has led AXIS Consulting to its position as the dominant provider for submissions to the Irish authorities and is now leading the expansion of the business into the UK, providing robust HTA submissions to NICE and SMC.
Speakers
Andrée Bates, PhD
Eularis, London, United Kingdom
Dr. Andrée Bates is a leading Artificial Intelligence thought leader and the founder and CEO of Eularis, a firm focused on tackling pharma industry challenges with artificial intelligence since 2003. She has worked in this space for pharma in Europe, USA, Canada, LATAM, EMEA, Japan, UK and China for over 20 years and in pharmaceuticals for 30 years. Dr. Bates has led Artificial Intelligence programmes for numerous top-tier pharmaceutical companies in diverse areas such as R&D, clinical trials, medical affairs and sales and marketing. These have all resulted in measurable efficiency and growth in profit and market share for her clients. Part of her success with AI is due to the initial strategic planning of AI across healthcare companies' value chain from a business perspective to ensure that pharma leaders can leverage the value of AI effectively to solve their unique growth and efficiency challenges successfully.
She is also a guest lecturer in 6 University MBA programmes in Health Innovation and AI and is also an Assessor for Innovate UK, UK’s national innovation agency, with her area of specialty being AI-focused start-up grants. She also hosts a podcast ‘AI for Pharma Growth’ that explores AI that is relevant to pharma.
Steve Beard, BSc, MSc
AXIS Healthcare Consulting Ltd, Dublin, Ireland
Steve has spent the last 27 years crafting his career as a seasoned HEOR / HTA / Market Access professional. This started with an academic position at the newly formed ScHARR, University of Sheffield, which was then followed with commercial business leadership positions in three successfully growing independent HEOR / Market Access consultancies: Global Head of HEOR, RTI Health Solutions, Chief Business Development Officer at BresMed Health Solutions, and Chief Consulting Officer, MAP Patient Access.
A common thread running throughout his career has been providing strong leadership and driving professional development for technical and commercial consulting teams, with a key focus on establishing and maintaining close and effective collaborative partnerships with industry clients. This is built on a firm belief that meaningful and effective collaboration in the consulting space can only come from a consistently applied philosophy of empowerment with highly functioning teams.
With a switch to independent consulting role, and a relocation to the Isle of Anglesey, Wales, Steve now provides HTA / HEOR strategic and technical insight to clients on a range of project types. These including HTA submission and payer / reimbursement applications. Steve also provides support to organisations / teams in planning for and navigating periods of organisational growth, the implementation of new working practices/change processes, and the management of periods of specific senior skill shortages.
When not working Steve can be found walking his new springer spaniel Milo on the beach, playing hockey for Eirias Hockey Club, and trying to establish some level of order to the woodland behind his house.
Dawn Lee, MMath, MSc
University of Exeter, Exeter, DEV, United Kingdom
Dawn is an Associate Professor of Health Economics and Health Policy, working with the Peninsula Technology Assessment Group (PenTAG), one of 11 research units in the UK providing expert advice on the clinical and cost-effectiveness of new drugs to the National Institute of Health and Care Excellence (NICE). She is also an existing member of the NICE interventional procedures appraisal committee.
Dawn joined PenTAG / Exeter University in Sept 2022 following over 15 years in economic consultancy where she worked most recent as the Chief Scientific Officer for a medium sized health economics consultancy (Lumanity; formerly BresMed). She is a health economic modeler who has conducted over 50 UK Health Technology Assessment submissions and worked in over 30 countries globally, a member of NICE’s interventional procedures advisory committee (IPAC) and the R for HTA group. Dawn’s key project achievements include representing manufacturers at ~30 HTA Committee meetings, working on the first ever immune-oncology submission to NICE (TA268), first ever PD-1 submission to NICE (TA384) and a considerable number following this and the first ITC accepted by G-BA.
Dawn’s main research interests are oncology modelling; particularly immune-oncology and therefore flexible survival modelling and the incorporation of external data within extrapolations, structured expert elicitation and improving diagnostic pathways.
Dawn is currently working on NICE's pathways pilot.
11:45 - 12:45
Member Group Meetings
ISPOR Europe Health Policy Update: Impacts of the EU Pharmaceutical Legislation Reform and the HTA Regulation on CEE Countries
Hosted by the ISPOR Central and East Europe (CEE) Consortium, this meeting will present timely updates of health economics and health policy in Europe. The session will discuss the reform of EU pharmaceutical legislation and the EU regulation of HTA by focusing on how these EU-wide collective efforts will impact CEE countries. What do these changes mean for CEE countries in terms of realigning health systems, contexts, and key players? How should CEE prepare for implementing policy reforms and what are the main challenges affecting implementation? Speakers will present unique perspectives and insights, highlight key issues, and provide recommendations on practical implementation for CEE. A Question and Answer (Q & A) section will be offered at the end of presentations.
Moderators
Oresta Piniazhko, PhD
State Expert Center of the Ministry of Health of Ukraine, Kyiv, Ukraine
Oresta Piniazhko, PhD, Director of HTA Department at State Expert Centre of Ministry of Health, Ukraine.
Oresta is an experienced expert in HTA, pharmaceutical policy and implementation practitioner. She holds a PhD degree in Pharmacoeconomics and is currently holding a position of Director of HTA Department at the State Expert Center of the Ministry of Health of Ukraine, ensuring management and implementation of the best international practices of HTA into health care system of Ukraine since February 2019. Being a dynamic communicator she is also a President of Ukraine ISPOR Chapter since 2017 and before ISPOR Ukraine Students Network (2015-2017). Oresta is visiting lecturer at The Institute of Business Education of Vadym Hetman Kyiv National Economics University and senior lecturer at Danylo Halytskyi Lviv National Medical University.
Speakers
Iga Lipska, MD, PhD, MPH
Health Policy Institute, Medical University of Gdańsk, Gdańsk, Poland
Iga Lipska is a Medical Doctor by education with Master of Science in public health. She graduated from the University of Utrecht in the Netherlands, where she defended her PhD thesis on health technology assessment of new medicines.
Iga has broad international and Polish experience in the healthcare sector, with a main focus on HTA at national and hospital level, drug pricing and reimbursement, health insurance, public health and evidence-based decision making process in health care.
She contributed her efforts significantly to the role of HTA in evidence based decision making processes on pharmaceutical reimbursement in Poland. She played a major role in HTA capacity building in Poland supporting the establishment of Polish national HTA Agency.
She also contributed her leadership and networking skills to the implementation of HTA in Central Eastern Europe and internationally advising in e.g. Croatia, Moldovia, Bulgaria, Turkey.
She served in variety of managerial roles in public and private sector supporting evidence based decision making in health care in Poland and abroad. She was the Director of Drug Policy and Pharmacy Department at Ministry of Health, the Director of HTA Department at Polish HTA Agency and the Director of Control Department at National Health Fund (public payer in Poland). She negotiated pharmaceutical reimbursement prices and Risk Sharing Schemes on behalf of Minister of Health while being the Economic Committee Member. She supported the establishment of global HTA benchmarking program at Center for Innovation in Regulatory Science in London, UK.
She has been co-chairing ISPOR HTA Roundtable Europe for 3 years’ term (from 2019 to the end of 2022).
Starting June 2022 she has been co-chairing HB-HTA Interest Group at Health Technology Assessment international for 3 years’ term.
Recently she has been affiliated to Medical University of Gdańsk which is also her Alma Mater.
She has just been nominated the Chairwoman of the Board at Health Policy Institute in Poland with her main role to contribute to evidence based policy in health care and build capacity in that area based on her international and national experience.
Momir Radulović
Agency for Medicinal Products and Medical Devices (JAZMP), Ljubljana, Slovenia
Medical Devices and Diagnostics Special Interest Group Open Meeting
Stop by to meet several members of the Medical Device and Diagnostics Special Interest Group’s (MD&D SIG) Leadership Team! We are hosting an open meeting to discuss many of the rapidly growing and complex challenges of medical devices and diagnostic technologies.
During our open meeting, we will discuss areas where we, the MD&D SIG Leadership Team, are focusing today, our pipeline of activities into the future including a recent publication in Value in Health , and how to stay connected with ISPOR and the SIG via our online community. Most importantly, we want to hear from you! What topics and areas interest you and where can we continue to provide value to your workstream?
The goals of the MD&D SIG are:
Develop and disseminate best practices in outcome research of medical technologies
Promote scientific discussions on relevant topics for devices and diagnostics
Prioritize, develop and promote research and education related to medical devices and diagnostics
Provide an international network of stakeholders to advance the validation, adoption and utilization of medical devices and diagnostics
We look forward to meeting you!
Educational Symposia
Defining Affordability and Sustainability to Revolutionize Cancer Care
Digital Conference Pass
Outcomes for cancer patients in Europe have improved significantly in recent years. But diagnoses are on the rise, due to factors including an ageing population and changing lifestyles. This puts increased pressure on health-care budgets and resources that are already overextended. Though the affordability and sustainability of cancer care are under constant scrutiny, disparate definitions and performance indicators have resulted in disjointed care pathways across Europe’s health-care systems. If innovation and best practices from European countries and their health-care stakeholders are to transform patient care through the redesign of cancer services, shared standards must first be established. Defining and measuring affordability, accessibility and sustainability in cancer care is an ambitious undertaking, and one of considerable importance given that cancer will affect around half of people during their lifetime. In contributing to that endeavour, this session will examine questions including:
How should we define and measure affordability and sustainability for cancer care in
What role will various stakeholders play in the definition and analysis of these factors?
What are the main shortcomings of cancer care in the areas of financial accessibility and
How can we draft and implement cross-border performance indicators for use in
Following a 15-minute presentation from our expert researchers at Economist Impact on the health system sustainability for oncology in Europe report, we welcome an audience-led Q&A session before beginning the discussions of this expert panel.
The 45-minute panel discussion will unite major stakeholders from the pharmaceutical industry, academia, associations for health-care professionals, patient organisations and government to discuss how to define the main factors in access to cancer care and what performance indicators should be used to assess its effectiveness.
Sponsored by BeiGene
Sponsor
BeiGene
Moderators
Gerard Dunleavy, PhD
Economist Impact, Dublin, Ireland
Gerard Dunleavy is a manager in the health policy team at Economist Impact. He manages global engagements with international clients in the health-care sector, from conceptualising to delivering and executing customised research projects. He manages multidisciplinary teams, conducting both quantitative and qualitative analyses, across a broad range of disease areas and health policies.
Prior to joining Economist Impact, Mr Dunleavy worked in academic settings specialising in evidence-based synthesis and epidemiological studies. He holds a PhD in public health and epidemiology and a master’s degree in health education and promotion, both from Maastricht University.
Speakers
Rifat Atun, Prof., PhD
Harvard University, Cambridge, MA, USA
Rifat Atun leads the Health Systems Innovation Lab at Harvard University, where he is professor of global health systems. His research focuses on health-system performance, health-system transformation and innovation globally. Dr Atun has advised governments around the world on health-system reforms and is a consultant and adviser to the World Bank, World Health Organisation and leading health and technology companies. In 2019-20 he was a senior adviser to the G20 presidency, and in 2021-22 he co-chaired its T20 Task Force on Global Health Security and Covid-19. From 2008 to 2012 he was director of strategy, performance and evaluation at the Global Fund to Fight AIDS, Tuberculosis and Malaria.
Simone Boselli, MSc
Eurordis, Rare Diseases Europe, Brussels, Belgium
Simone Boselli joined EURORDIS in 2017 as public affairs director. A member of the European and international advocacy team, he contributes to policy development and represents EURORDIS in discussions on access to therapies, with a focus on reducing delays and inequalities, on the underlying challenges in the field of value assessment, pricing and reimbursement of orphan medicines, and on initiatives towards improved access. Mr Boselli contributed to EURORDIS’s position paper on “Breaking the Access Deadlock to Leave No One Behind”, and he supports advocacy activities at Rare Diseases International and the development of the NGO Committee for Rare Diseases.
Guillaume Dedet, MD, MPH
Organisation for Economic Co-operation and Development (OECD), Paris, France
Guillaume Dedet is senior health economist at the OECD Health Division, where he leads a project on inequalities in cancer care and prevention in the European Union, as well as the OECD/EU’s regular cycles of comparative analysis of health systems and the health of populations across Europe. Dr Dedet is a lecturer at SciencesPo Paris and is secretary of the French Public Health Association (SFSP). Prior to joining the OECD he was a medical adviser at the French Ministry of Health, and a technical officer at the World Health Organisation Regional Office for Europe. He was the 2022-23 French Harkness fellow in health-care policy and practice at Stanford University.
Shreya Jani, MPH
BeiGene, New York, NY, USA
Shreya Jani joined BeiGene in 2021 and is senior vice-president of corporate affairs. She directs the company’s integrated approach to patient engagement and public-health policy, as well as social responsibility, communications and media relations. Ms Jani has a deep background in health-care policy and in corporate and reputation communications. Before joining BeiGene she led corporate affairs for Pfizer’s vaccines, oncology and consumer health-care businesses, and counselled chief executives on communications, reputation, policy and public affairs. Earlier in her career Ms Jani worked for Margaret Sanger International and the World Health Organisation in Geneva, where she focused on HIV/AIDS and reproductive-health issues.
EuHTA Impact on Innovations: Expectations and Challenges of EuHTA for Germany
Digital Conference Pass
With the new EU HTA Regulation, the benefit assessment of new therapies is regulated for the first time at the European level. It regulates a joint clinical assessment of new medicinal products at European level, which is to start from January 2025 for the first products, including advanced therapy medicinal products (ATMPs) and oncology medicinal products (incl. orphan drugs). The framework is to be concretised. We want to discuss with several stakeholders from medical societies, health insurances and industry challenges and opportunities of EuHTA.
Sponsored by Kyowa Kirin GmbH
Sponsor
Kyowa Kirin GmbH
Speaker
Eva Susanne Dietrich, MD
Institute for Evidence-Based Positioning in the Healthcare Sector, Bonn, Germany
Professor Eva Susanne Dietrich is a pharmacist with more than 20 years professional experience in reimbursement, health politics, and drug evaluation.
She graduated at the University of Heidelberg, obtained her PhD in the field of pharmacoeconomic methodology from the University of Tuebingen and received a Master’s degree on Health Technology Assessment and Management from the University of Barcelona.
Professor Dietrich served as a deputy member of the Federal Joint Committee (G-BA) which specifies for almost 90 percent of the German population the services in medical care that are reimbursed. In parallel, she led the Department of Drugs, Remedies and Aids at the German National Association of Statutory Health Insurance Physicians (KBV) which concludes contracts with health insurance funds and other parties of the health care sector on the part of the 140,000 office-based physicians and psychotherapists in Germany. In the following years, she organized a scientific institute for Techniker Krankenkasse, one of the largest health insurance funds in Germany, and was responsible for the Management Division Health Sciences. Thereafter, she managed a scientific consulting firm in Basel for eight years and founded the Institute of Evidence-based Positioning in the Healthcare Sector in 2018.
An important emphasis of her professional and academic activities is the critical review of the evidence of new drugs, their classification in the existing care context and, more specifically, early benefit assessments and the German AMNOG process as well as the compilation of AMNOG dossiers.
Since 2000, Eva Susanne has been teaching at the department of clinical pharmacy at the University of Bonn, where she holds a honorary professorship and also leads a module in the drug regulatory affairs degree programme. Apart from this she taught pharmacoeconomics, evidence based medicine, and benefit legislation e.g. at the universities of Hamburg, Berlin and Marburg.
Thomas Ecker, PhD, MA
Accessus Health GmbH, Hamburg, Germany
Lutz Herbarth, MBA
KKH Kaufmännische Krankenkasse, Hannover, Germany
Julia Rumsch, LL.M.
Federal Association of the Pharmaceutical Industry (BPI), Brussels, Belgium
After studying law in Germany, Julia Rumsch has worked in the life science sector in Germany, the UK, and Brussels. Since 2017 she has worked for the German Pharmaceutical Industry's (BPI) liaison office in Brussels with a strong focus on European health care and industrial policies.
Discussion Groups
Embracing the AI Revolution: Exploring the Promising Future of Generative AI in HEOR
Moderator
Bill Malcolm, MSc
Bristol Myers Squibb, Middlesex, LON, United Kingdom
Bill Malcolm leads the Economic & Predictive Modeling group within the Advanced Scientific Capabilities function of global HEOR for Bristol Myers Squibb. Bill has worked across a host of therapeutic areas and roles in pharma over a period of 21 years.
EU Joint Clinical Assessment – One for All and All for One?
Moderator
Anke van Engen, MSc
IQVIA, Amsterdam, NH, Netherlands
Anke is a Managing Principal and leads the HE/HTA Center of Excellence in the European Value & Payer Evidence practice. She has over 20 years of experience and has co-authored many articles and spoken at international conferences.
ISPOR Forums
Are We Patient-Centered Yet? Patient Engagement and Patient-Centered Research in HEOR
Including patients as partners to ensure research meets the needs of the population being studied is now strongly encouraged by regulatory agencies and funding bodies. Sponsors and researchers often describe their research as patient centered, but it is not clear how or if patients were actually engaged. The ISPOR Patient-Centered Special Interest Group (PC SIG) invites all stakeholders (e.g., patient centered, oncology, COA, health preference, and HEOR) from the ISPOR community to learn about opportunities for patient engagement across stages of study development and in various study types.
In this session, speakers will define patient centricity and engagement; offer strategies for engagement for a range of HEOR study types and contexts; and identify practical and scientific challenges to engagement as well as possible solutions. We will discuss key considerations including engagement when resources may be limited, representative engagement and working with underrepresented groups.
Dr Roydhouse will provide an overview of the topic and the PC SIG’s previous and current work regarding patient engagement. Dr Escontrias will discuss patient engagement and the engagement of underrepresented groups from a patient advocate perspective. He will also describe the numerous resources the National Health Council has developed over the past several years on meaningful patient engagement. Dr Carlton will discuss patient engagement in patient-reported outcome measure development and value/HTA studies (10m).
Polling will be used to capture audience experiences with patient centricity/engagement. Twenty minutes will be reserved for moderated audience discussion, including a Q&A session.
As the PC SIG has developed a definition of engagement in research and is gathering data on the ISPOR community’s use of patient engagement strategies, this session is timely and can inform future SIG work, including contributing to a potential future publication.
Moderators
Jessica Roydhouse, PhD
Menzies Institute for Medical Research, Hobart, TAS, Australia
Dr Jessica Roydhouse is Select Foundation Senior Research Fellow in Health Services Research at the Menzies Institute for Medical Research, University of Tasmania, Australia. In this role she also serves as Director of the Tasmanian Cancer Registry. Her research interests include patient-reported outcomes, cancer clinical trials and missing data.
Speakers
Jill Carlton, BMedSci, MMedSci, PhD
University of Sheffield, Sheffield, United Kingdom
Jill is a senior research fellow in at the School of Health and Related Research at the University of Sheffield, UK. Jill’s research interests lie in measuring and valuing health, primarily through the development of patient reported outcome and preference-based measures. She is a mixed methods researcher who has led projects across numerous health conditions. This includes children and carers, as well as in rare, progressive life-limiting conditions. Jill has been involved in many projects which have had active stakeholder engagement, including developing a framework for fully incorporating public involvement (PI) into patient-reported outcome measures (PROMs).
Omar Escontrias, DrPH, MPH
National Health Council, Washington, DC, USA
Dr. Omar A. Escontrías’ expertise is centered in the areas of community and patient engagement, coalition building, health policy, and evidence-based research. Over the span of his career, Dr. Escontrías has worked with local and state governments, as well as non-profit organizations in the areas of public policy, disease prevention, and health promotion to improve the health and lives of patients and communities. By the time he joined the National Health Council (NHC) in April 2022 as its Senior Vice President of Equity, Research and Programs, he had already spent 15 years building a career on fighting systemic health care inequalities in historically underrepresented communities. Dr. Escontrías is a proud first-generation immigrant and is thrilled to be contributing to the NHC’s patient-centered advocacy, health equity, and policy engagement efforts. Dr. Escontrías earned a Bachelor of Science in molecular and cellular biology, a Master of Public Health in epidemiology, and a Doctor of Public Health in health policy and management, all from the University of Arizona.
Assessing Real-Word Data Quality From Electronic Health Records for HTA: Good Practices Task Force Preliminary Recommendations
The increasing availability of electronic health records (EHR) provides new opportunities to improve and refine health technology assessments (HTAs). One of the most compelling aspects of EHR-derived real-world data (RWD) is their ability to define real-world patient populations with high degrees of clinical specificity relative to other real-world databases, such as insurance claims or registries. EHRs capture most major clinical factors that clinicians consider when making therapy choices in practice, as well as diagnostic result information that guides decision making.
However, there are issues that are unique to EHR-derived RWD that must be considered to ensure that evidence generated from them is suitable for decision making. Moreover, there are no agreed upon data quality guidelines for including EHR-derived data in evidence reports or for HTA groups reviewing those reports. To address this gap, ISPOR established a task force to develop emerging good practices for the use and interpretation of EHR-derived RWD for HTA.
During the presentation, speakers will summarize the task force’s framework for understanding the suitability of EHR data for HTA. The framework adopts a proportionate risk-based approach that depends on the use case and decision context. Key elements include data provenance and data fitness for purpose, the latter focusing on reliability and relevance for decision making. Panel members will present the ISPOR Suitability of EHR Data (SUITED) Checklist for HTA Evidence Developers, and proposed policy recommendations to help improve the usability of EHRs for HTA bodies and other decision makers.
Following a discussion of research priorities, the panel will host a Q & A session on the checklist and recommendations. Presented by the ISPOR Use of Electronic Health Records (EHRs) in HTA Task Force
Moderators
Scott Ramsey, MD, PhD
Fred Hutchinson Cancer Center, Lake Forest Park, WA, USA
Dr. Scott Ramsey is a Professor at Fred Hutchinson Cancer Center and the University of Washington. He directs the Hutchinson Institute for Cancer Outcomes Research, a multidisciplinary team devoted to clinical and economic evaluations of cancer prevention, screening and treatment.
His research portfolio includes SEER-Medicare/Cancer Registry data linkages, patient outcomes, economic modeling of interventions, cost-effectiveness analysis, quality of life assessment, health care utilization, economic burden, pragmatic trial design, early technology assessment, and stakeholder engagement.
Speakers
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is an epidemiologist and economist accelerating the development of valuable medicines and health policy. She serves as Principal Scientist at Flatiron Health in New York. Dr. Adamson advances artificial intelligence applications of natural language processing and machine learning in the curation of electronic health records for real world evidence with a team of engineers, scientists, and clinicians. She designs treatment comparative-effectiveness studies, codes mathematical models of disease, and delivers research for global health technology assessment. Dr. Adamson served as a former member of the White House COVID Task Force on Healthcare Resilience while lead pandemic data scientist in the West Wing. She is an Affiliate Professor at the University of Washington in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute. Dr. Adamson has held roles at the Bill and Melinda Gates Foundation Institute for Disease Modeling, Infectious Economics, the NIAID HIV Vaccine Trials Network, and Fred Hutch Cancer Center. She continues to serve as a leader in the International Society for Health Economics and Outcomes Research (ISPOR). Dr. Adamson holds degrees in pharmaceutical economics, public health epidemiology, and microbiology. She enjoys writing about the economics of infectious diseases at www.blytheadamson.com.
Seamus Kent, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Seamus is Assistant Professor in HTA at Erasmus University Rotterdam. Prior to joining Erasmus Seamus was a Senior Adviser in Data & Analytics at NICE where he led the development of NICE's Real-world evidence framework and a Senior Adviser in HTA at Flatiron Health a RWD company in oncology.
Patrick Muller, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Patrick Muller is a Technical Adviser (Data Science), in the Methods team in the Centre for Guidelines at NICE. He advises on research methods and leads RWE primary analysis projects within the Centre. Previously he worked as research fellow in epidemiology at LSHTM and before that as a trial statistician at UCL. He has a PhD in epidemiology, an MSc in medical statistics, and a BSc in Mathematics.
Innovative Funding and Financing Models for Non-Communicable Diseases (NCD) in Low- and Middle-Income Countries in MENA Region
Health systems in the Middle East and North Africa (MENA) are striving to balance healthcare funding and rising costs in response to the substantial health burden posed by non-communicable diseases (NCDs). The task becomes even more challenging in the wake of economic turmoil such as those witnessed in Egypt and Lebanon. Concurrently, countries from North Africa like Morocco are looking to ensure long-term sustainability by establishing Health Technology Assessments (HTAs) aimed at optimizing spending and looking into innovative funding mechanisms for NCDs. Regardless of the driving factors, nations in the region are exploring novel methods for healthcare financing, such as blended financing models, multisource crowd funding, novel private insurances, Public Private Partnership, and others. What are the challenges and opportunities associated with financing non-communicable disease (NCD) care in resource-constrained settings? How can innovative approaches address these challenges? And how applicable are these approaches to low- and middle-income countries (LMICs) in MENA? This webinar convenes experts to exchange experiences and assess the feasibility of new financing models. Dr. Amal Yassine will discuss the challenges related to regulating expenditures associated with NCDs in Morocco and explore a new financing model that can support the control and management of these conditions while considering therapeutic progress and improved access to healthcare innovation. Dr. Wadih Mina will explain the impact of the financial crisis on the Lebanese health sector, including the unaffordability of healthcare for the majority of the population, the government's response to the crisis, a draft law for Universal Health Coverage (UHC) proposing new funding channels, and critical success factors for implementing innovative funding models based on the Lebanese experience. Dr. Mahmoud El Mahdawy will provide a focused analysis on Egypt, addressing the challenges and opportunities related to financing NCDs, exploring innovative approaches, and assessing their applicability within the Egyptian context.
Moderators
Rita Karam, PharmD, PhD
Lebanese University, BEIRUT, BA, Lebanon
Rita Karam is a Pharmacist graduated from Saint Joseph University of Beirut, Lebanon.
She holds a Ph.D. in Pharmaceutical Sciences from Claude Bernard University Lyon -France.
She is a Professor at the Faculty of Sciences and Medical Sciences at the Lebanese University.
She is the Director of Quality Assurance of Pharmaceutical Products Program and the National Pharmacovigilance Program Coordinator.
She is a member of ISoP (International Society of Pharmacovigilance),
She is the Chair of the Professional Society for Health Economics and Outcome Research ISPOR -Arabic Network (Chair of ISPOR-Arabic Network)
She technically advises several committees at the Ministry of Public Health.
She has authored numerous articles in the field of Health Technology Assessment, Health Economics and Pharmacovigilance
Speakers
Mahmoud Elmahdawy, PharmD
Novartis, Basel, Switzerland
Mahmoud Elmahdawy, is a PharmD graduate from Drake University (USA), College of Pharmacy, May 2001. Mahmoud Has over 20 years of experience in Academia, Regulatory authority and Industry.
In July 2010, Dr. Elmahdawy accepted a challenge and moved to the Egyptian Drug Authority (EDA) as general manager of Hospital Pharmacy Administration. His department oversees procurement, Clinical Pharmacy and Pharmacoeconomics.. The department is responsible for clinical pharmacy fellowship program, and supervises clinical pharmacy implementation across the government hospitals in Egypt.
Dr. Elmahdawy is the president of International Society of Pharmacoeconomics and Outcomes Research (ISPOR) Egypt’s chapter, which was established in July 2011. Mahmoud is the past president of ISPOR Arabic network. Dr. Elmahdawy has lectured and moderated several Pharmacoeconomics conferences and workshops. As part of a team, a Health Economics postgraduate diploma was launched for the first time in the Middle East Where Dr. Elmahdawy was amongst the first graduating wave. Proudly, ISPOR Egypt’s chapter is amongst active chapters with dynamic activities and helping to further spread Pharmacoecomics awareness across the region.
Mahmoud joined Novartis Patient Access team in 2015 and was located in Singapore (2018-2022) as Global Pricing and Access Director. Mahmoud is currently Executive Director for Hematology Policy at Novartis and is based in Basel, Switzerland.
Mina Wadih, PharmD, MSc
Pharma International Roche, Beirut, Lebanon, BA, Lebanon
Wadih Mina is a Lebanese Pharmacist, Holding a MSc in Pharmaceutical Marketing, a MSc in health Economics, and currently completing his MSc in Global Health Policy.
Wadih is currently assuming the role of Health Systems & Policy Lead for the Levant Region at Roche.
Wadih is also the president elect of the ISPOR Lebanon Chapter, and the co-founder of the Webseries Health Policy Corner.
Amal Yassine, MD, PhD
National Health Insurance Agency, Mohammedia, MOH, Morocco
Dr Amal Yassine - Morocco
General Practitioner
PhD in life and health sciences
Head of division of the agreement division at the National Health Insurance Agency (ANAM) in Morocco with 14 years of professional experience in health financing
Temporary teacher at the Mohammed VI University of Health Sciences in Casablanca & Faculty of Medicine, Mohammed V University in Rabat, Morocco
Member of the executive committee of Moroccan Society of Health Products Economy (SMEPS) as secretary general, Morocco
Member of the executive committee of the Latin Association for the Analysis of Health Systems (ALASS) – Spain
Member of ISPOR Maghreb Chapter.
12:15 - 12:45
Exhibit Hall Theater
An Innovative Multi-Comparator Approach to Cost-Effectiveness Analysis Designed to Reflect the Real-World Value in a Crowded Market
Digital Conference Pass
Determining the most appropriate comparator for use in cost-effectiveness analyses is one of the critical decisions that need to be made when developing economic evidence for reimbursement or payer decisions. While a standard cost-effectiveness analysis may include features to allow for the user to switch between the comparator of interest within the model, this remains a one-vs-one comparison.
However, while using one comparator for an ICER may be the standard approach, it will not necessarily demonstrate the true value of a product launching into a marketplace which may have multiple existing therapy options, since these will not all necessarily be displaced by the new therapy. For healthcare professionals, the decision is often not a simple displacement of the most efficient existing comparator to the new intervention for all patients of interest, but there is a need to consider a range of different aspects for different patients which may result in a variety of approaches. Therefore, to optimally inform reimbursement decisions, understanding how the efficiency of clinical practice is changed by the inclusion of an additional treatment option within the marketplace as whole, is critical.
This theatre presentation will present an innovative framework for multi-comparator cost-effectiveness analyses, the Adelphi Values PROVE™ PACE™ framework, which allows for analysis of the effect a new treatment would have across clinical practice.
Sponsored by Adelphi Values
Sponsor
Adelphi Values
Speaker
Louise Heron, MSc
Adelphi Values PROVE™, Bollington, CHE, Great Britain
Louise drives innovation and growth across our team. With oversight across all client delivery, she provides senior strategic input to optimize market access for new products. She has extensive experience in leading projects to gain insight into product value, reimbursement opportunities, and in developing health economic models and payer funding tools. She holds a degree in Natural Sciences from the University of Cambridge and an MSc in Economic Evaluation for Health Technology Assessment from the University of York.
Robert Hughes, MSc
Adelphi Values PROVE, Bollington, CHE, United Kingdom
Robert is a Senior Health Economist in the PROVE™ practice within Adelphi Values. Robert is one of the senior technical leads in the team and oversees a range of health economic and data analysis projects, as well as providing mentorship, training and providing direction for the rest of the Health Economics and Data Analysis team. He also contributes to wider projects and strategic discussions generate the most appropriate market access solutions. Robert has worked across a range of different therapeutic areas, including having extensive experience in oncology modelling, and has developed a number of innovative modelling frameworks that help to expand the model’s scope beyond that of the traditional modelling approaches. Prior to joining Adelphi Values, Robert gained experience in Health Economics research within the Centre for Health Economics at Monash University, producing results that have helped inform health policy decisions in Australia. He holds an honours degree in Economics from Newcastle University and an MSc in Health Economics from the University of York.
12:15 - 13:15
Special Onsite Join/Renewal Event for ISPOR Membership
Special Onsite Promo! Stop by the ISPOR Booth to join or renew your ISPOR membership! We might just have a surprise for you...
12:45 - 13:15
Exhibit Hall Theater
Using AI-Enriched Electronic Health Record Data to Access an Additional 75% of Data to Drive Real-World Evidence and Trial Recruitment in Mental Health and Dementia
Digital Conference Pass
Akrivia Health curates a database of 4.5m patients’ deidentified electronic health records from NHS secondary care psychiatric healthcare organisations, enriched with a bespoke, AI-based natural language processing pipeline (Akrivia Synapse™). This presentation will explore how we use this uniquely rich and demographically diverse dataset to generate real world evidence (RWE) and drive recruitment for prospective research and clinical trials. Understanding how to effectively utilise this powerful data resource can allow critical research questions in mental health and dementia to be addressed, and can streamline patient recruitment for clinical trials.
In this session, we will examine how Akrivia Health is supporting HEOR activities by deploying large scale, AI-enriched real-world data to deeply characterise patient populations, monitor treatment effectiveness, and map care outcomes.
Sponsored by Akrivia Health
Sponsor
Akrivia Health
Moderators
Tina Marshall, N/A
Akrivia Health, Oxford, United Kingdom
Tina is a highly accomplished and experienced commercial leader who has made a significant impact in the medtech sector for over 25 years. Having worked with renowned organisations such as Becton Dickinson, KCI Medical (3M), and ICNet (Baxter), Tina possesses a wealth of expertise in driving success with revenues ranging from £1M to £35M. She has a proven track record of launching new businesses, building commercial capability, and achieving impressive first sales. As a dynamic leader, Tina takes charge of the end-to-end customer journey, leading Sales, Marketing, and Customer Success. Her unwavering passion for driving innovation in healthcare has fueled her success in leading teams, expanding markets, and maximising revenue growth. With her strategic thinking, exceptional business acumen, and profound industry knowledge, she excels in navigating complex regulations and identifying emerging trends.
Tina firmly believes in the power of collaboration, individual empowerment, and creating inclusive work environments. She actively advocates for women in leadership, recognising the value of diversity and its role in driving innovation and improving patient outcomes.
Speakers
Benjamin Fell, PhD
Akrivia Health, Oxford, United Kingdom
Ben heads up Akrivia Health's Research & Development team. His academic background is in experimental psychology but has worked for the past 5 years on the application of real world administrative data to improve research, policy and frontline services. At Akrivia, Ben leads the AI team's natural language processing development program, and oversees data science activities for our clinical trial support and consultancy services. Ben's personal research interests include the use of real world data to bridge translational gaps in mental health research, social determinants of health and health inequalities, and transparency and privacy-enabling application of AI.
13:15 - 14:15
Member Group Meetings
ISPOR Korea Chapter Meeting
The ISPOR Korea Chapter extends a warm welcome to anyone interested in connecting with us. Established in 2010, the chapter currently boast a great number of members. We invite you to join and discover what’s happening in the HEOR field in Korea.
ISPOR Good Practice Task Force Recommendations on Valuing HRQoL of Children & Adolescents in Economic Evaluation Open Meeting (Pediatric Utilities)
Methods guidelines for economic evaluation by international HTA agencies lack guidance specific to child HRQoL utilities, or implicitly assume that what is recommended for valuation of adult HRQoL is also appropriate for children and adolescents. Yet there are a number of methods issues specific to the valuation of HRQoL in children that do not arise in the valuation of adult HRQoL. The methods used to preference-weight pediatric HRQoL instruments differ in important ways that decision makers may not be aware of.
There are important gaps in both methods research and guidance from decision makers about pediatric HRQoL utilities for use in economic evaluation. ISPOR launched Good Practice Task Force on pediatric utilities to address these issues. This forum will highlight the principal methods issues concerning:
Whose stated preferences are relevant to the preference weighting of pediatric HRQoL states?
What perspective respondents should be asked to adopt in imagining and valuing pediatric HRQoL?
What preference elicitation methods should be used? and
To what extent is the comparability and consistency of health state utilities for adult and child HRQoL important for their use in QALY estimation, economic evaluation and decision making?
Audience feedback will be sought on the overall approach to the identification of good practice in pediatric utilities including our preliminary recommendations. Good practice concerning pediatric utilities inevitably involves both HEOR science and normative considerations. Given the pivotal role of value judgements in methods choices concerning pediatric utilities, consultation with local HTA bodies is essential both to ensure that utilities are ‘fit for purpose’, and to ensure that decision makers are well informed about the role and implications of value judgements when establishing methods guidelines. We conclude by identifying evidence gaps where further research is required to improve the scientific basis for pediatric utilities.
Moderators
Donna Rowen, MSc, PhD
University of Sheffield, Sheffield, United Kingdom
Donna Rowen is Professor of Health Economics at the University of Sheffield in the UK. Her
research interests lie in the measurement and valuation of health to inform health technology assessment.
Speakers
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Senior Fellow at the Office of Health Economics, London. She is Chair of the Board of the EuroQol Research Foundation and served as President of ISPOR (2019-2020). Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money; and priority setting in health care. She is currently leading a large programme of research to improve measurement and valuation of quality of life in children (QUOKKA), funded by the Medical Research Future Fund (Australia). Nancy has published >180 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.
Louis Matza, PhD
Evidera, Bethesda, MD, USA
Elly Stolk, PhD
EuroQol Research Foundation, Rotterdam, Netherlands
Elly Stolk is Scientific Director at the EuroQol research foundation. Elly also is founding co-director of the Erasmus Choice Modelling Centre (ECMC). Her main areas of expertise are measurement and valuation of health and assessment of quality of life using patient reported outcome measures (PROMs). Her core responsibilities are managing the EuroQol research funding program, high quality project delivery, and to provide support and guidance to Office team members and other researchers. She has over 100 publications in this area.
13:45 - 14:45
Spotlight Session
Navigating Challenges and Seizing Opportunities: Leveraging Multiple RWD Sources in External Control Arms for HTA and Regulatory Decision-Making
Digital Conference Pass
Level: Introductory
ISSUE: In the absence of randomized controlled studies, especially for oncology and rare diseases, external control arms (ECA) using real world data (RWD) may be executed to provide context to single-arm trial findings in support of health technology assessment (HTA) and regulatory submissions. To address the challenge of limited sample size, multiple RWD sources such as chart reviews, electronic health record (EHR) databases, registries, and clinical trial extensions are increasingly utilized together to strengthen ECAs. Leveraging multiple RWD sources also offers valuable insights into treatment effects across diverse populations, geographies, data types, and timeframes. However, challenges persist in aligning trial criteria, treatment modalities, endpoints, and confounders across different data sources. As personalized medicine gains prominence and the demand for valid and successful ECAs grows, it is crucial to discuss the challenges and approaches to harmonizing multiple RWD sources for ECA creation.
OVERVIEW: This panel aims to explore how leveraging and harmonizing multiple RWD sources can enhance the evidentiary requirements of global HTA and regulatory bodies. Moderated by Dr. Chris Cameron, the panel will provide an overview of HTA/regulatory guidance documents on RWD/RWE sources and summarize recent ECA submission review results.
Key questions for the panelists include:
What challenges and opportunities arise when harmonizing multiple RWD sources for creating ECAs in HTAs and regulatory submissions?
How can we address emerging RWE guidance and enhance acceptance of ECAs by harmonizing multiple RWD sources?
International experts from RWE research organizations, industry sponsors, and academia/HTA reviewers will contribute their perspectives. Topics of discussion will encompass designing valid and transparent ECAs using multiple RWD sources, industry sponsors' experiences with ECAs, appropriate use of causal inference methods from academic viewpoints, and utilizing ECAs for risk-benefit assessment. The target audience includes HTA agencies, regulators, academics, patient advocacy organizations, and pharmaceutical/biotech manufacturers.
NOTE: There will be a discussion group on this topic following the session. Join us in the Discussion Lounge in Hall E North from 15:15-16:15 in Discussion Group B.
Moderators
Chris Cameron, MSc, PhD
EVERSANA, Sydney , NS, Canada
Dr. Chris Cameron is Chief Scientific Officer at EVERSANA. He is a global thought leader in health economics and outcomes research with over 15 years of experience. Prior to joining EVERSANA, Chris was a partner at Cornerstone Research Group Inc., and lead of Health Economics at CADTH. He has also been a consultant decision scientist for ICER, a member of the Economic Guidance Panel at pCODR, and served on PMPRB Technical Panel on drug pricing guidelines in Canada. Chris is an adjunct professor at Dalhousie University, and has authored over 180 peer-reviewed publications in many of the world’s top journals, including JAMA, Lancet, and BMJ. His research has been featured in the Globe and Mail and New York Times. He was recently named one of the most prolific authors in the world on indirect treatment comparisons and was a co-author on the PRISMA NMA reporting guidelines. Chris holds a bachelor’s degree in mathematics and an MSc and PhD in Epidemiology where he was a Vanier Canada Graduate Scholar. He also completed a fellowship on real-world data analytics at Harvard University and FDA’s Sentinel Initiative.
Panelists
Nicolle Gatto, PhD
Aetion, New York, NY, USA
Dr. Gatto has over 20 years of pharmacoepidemiological and regulatory experience across industry and academia. She is the Chief Science Officer and leads the Scientific Research and Strategy team at Aetion, Inc, a technology company that provides analytic software and research services to the healthcare industry including biopharma, regulators, and payers. In this role, she serves as a senior scientific expert on methodological and regulatory-related studies across therapeutic areas, and partners with biopharma companies and decision-making bodies, such as regulatory agencies and Health Technology Assessment agencies (HTAs). Prior to Aetion, Dr. Gatto worked at Pfizer where she oversaw Vaccines and Oncology Epidemiology and led a team to design and implement post-authorization safety and effectiveness studies, and pre-approval studies to support development and regulatory decision-making across Pfizer’s portfolio. She is a Fellow of the International Society for Pharmacoepidemiology, and an Adjunct Assistant Professor of Epidemiology at Columbia University and Tulane, where she has taught courses in pharmacoepidemiology, causal inference and confounding control methods. She has a Bachelor’s degree in biology from University at Albany, State University of New York, a Master’s degree in Public Health from the New York University School of Education, and a Doctorate degree in Epidemiology from the Columbia University Mailman School of Public Health.
Qiufei Ma, PhD
Regeneron Pharmaceuticals, Inc, Oak Park, CA, USA
Qiufei Ma, PhD, is senior director HE&OR at Regeneron Pharmaceuticals, Inc. Dr Ma is passionate in optimizing evidence and data-driven decision for drug development & commercialization. He has fifteen years of experience in health economics, real-world evidence, market access & pricing, HTA submissions, and global product launches across multiple therapeutical areas/platforms including oncology, hematology, cell & gene therapies, biosimilar, and general medicine.
Uwe Siebert, MD, MPH, MSc, ScD
UMIT TIROL - University for Health Sciences and Technology, Hall in Tirol, Austria. ONCOTYROL - Center for Personalized Cancer Medicine, Innsbruck, Austria. Harvard T.H. Chan School of Public Health and Harvard Medical School, Hall in Tirol, 7, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a Professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), Chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL - University for Health Sciences and Technology in Austria and Director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also Adjunct Professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in Decision Sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE, EUCAPA, PREMIO COLLAB). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (>30,000 citations, H index >80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
Breakouts: IP, WS, & OBS
Which Novel Indirect Comparison and Network Meta-Analysis Methods Are Relevant for Health Technology Assessment Submissions?
Digital Conference Pass
Level: Experienced
ISSUE: Indirect treatment comparisons (ITC) and network meta-analyses (NMA) are a key element of many health technology assessment (HTA) submissions. Although best practices have been established, there has been a lot of methods research and development to further improve ITC/NMA methods in the last few years. In this session a selection of recent ITC/NMA trends and developments will be presented. We will debate whether these are actually targeting the biggest challenges given an increasingly complex evidence base, and whether these can be recommended for HTA submissions.
OVERVIEW: In the last few years many novel ITC/NMA methods have been proposed to ensure treatment effects are relevant for the target population, “disconnected networks” and single arm trials can be incorporated, survival data appropriately analyzed, and real-world evidence (RWE) utilized. The question is whether recent developments in ITC/NMA methods are needed, credible, and relevant for HTA submissions. After introducing the session, and providing a topline overview of recent trends and development in ITC/NMA methods (Di Maio, ~10 minutes), panel members will present a selection of key methods in more detail, including methods for population-adjustment (Dias), methods for disconnected networks (Thom), and NMA methods to analyze survival data and facilitate time-inhomogeneous Markov state-transition cost-effectiveness models (Jansen) (~8 minutes each). Subsequently, the panel members and audience will have a debate lead by the moderator (20 minutes). In addition to questions from the audience, the following questions will be discussed: 1) What are the biggest challenges to ITC/NMA given the increasingly complex evidence base for new interventions and the increased use of RWE? 2) Are recent ITC/NMA methods actually targeting these challenges? 3) Which of the recent methods are of relevance and can be recommended for HTA submissions; and 4) What further work is needed to make methods more acceptable and useable?
Moderators
Danilo Di Maio, PhD
F. Hoffman-La Roche, Basel, BS, Switzerland
Danilo Di Maio is a Global Access Evidence Lead at Roche. In this role, he led the development of evidence packages and contributed to several reimbursement submissions to HTA bodies worldwide in the areas of oncology, neuroscience and rare diseases. Prior to joining Roche, he worked in the consulting industry where he supported clients develop evidence and market access strategies for pharmaceuticals and diagnostics. Before joining the industry, he was in academia working on early stage pharmaceutical and biomedical research. Danilo holds a PharmD, a PhD in Molecular Modelling and a Master in International Healthcare Management, Economics and Policy at SDA Bocconi School of Management.
Panelists
Sofia Dias, PhD
University of York, York, YOR, United Kingdom
Sofia is Professor of Health Technology Assessment at the University of York (UK) and Director of the CRD/CHE Technology Assessment Group that delivers technology assessment reports for the National Institute for Health and Care Excellence (NICE) through York's NIHR funded TAR programme. Sofia is also a member of NICE Technology Appraisals Committee D.
She is a statistician with interests in Bayesian methods for evidence synthesis and their application to decision-making. Of particular interest are methods for network meta-analysis, bias-adjustment, synthesis of related outcomes and population-adjusted treatment comparisons.
Sofia has collaborated with the NICE Decision Support Unit to produce several technical support documents which provide guidance to all those involved in submitting or critiquing evidence as part of NICE Technology Appraisals and is lead author of the Wiley book on Network Meta-analysis for Decision Making. She also has a wide experience of developing and applying evidence synthesis methods in NICE clinical guidelines and Cochrane Reviews.
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Howard Thom, MSc, PhD
University of Bristol, Bristol, UK; Clifton Insight, Bristol, SOM, United Kingdom
Howard Thom is a Senior Lecturer in Health Economics at the University of Bristol and Senior Director at the consultancy Clifton Insight. His research interests are value of information, uncertainty in economic models, network meta-analysis, and R for health economics.
The WHO Europe′s Access to Novel Medicines Platform (NMP) Facilitating Access to High-Cost Medicines for Patients in Europe Through Multi-Stakeholder Engagement
Digital Conference Pass
Level: Intermediate
ISSUE: There have been major strides in biopharmaceutical research and advances in prevention, screening, diagnosis and treatment in recent decades, thanks to investment from the public and private sectors. However, all countries have voiced concern over the escalating prices and affordability of medicines and especially the budgetary impact of novel medicines. This has led to restricted patient access, increased inequities, and financial hardship when health systems are already under significant financial and fiscal constraints because of the economic context and socioeconomic recovery from COVID-19.
At the 72nd session of the WHO Regional Committee for Europe in September 2022, the WHO Regional Office for Europe, in agreement with Member States, issued a statement of intent to act as a neutral convenor for the establishment of a platform for collaboration, to improve patient access to novel high-cost medicines. The WHO Europe’s Access to Novel Medicines Platform (NMP) will enable Member States, non-State actors and other partners to coordinate, collaborate, prioritize and align efforts to deliver solutions to improve access for patients.
OVERVIEW: Tarang will introduce the NMP with its aims, objectives and technical areas and introduce the different stakeholders on the panel. 5 mins
Meindert will present the challenges NICE is facing in providing access to novel medicines to England and Wales, and what they hope to resolve through international cooperation. 10 mins Simone will present the challenges patients have in accessing orphan drugs and what possible solutions EURODIS would like to see move forward through the NMP. 10 mins Thomas will present the barriers industry is facing in providing access to novel medicines and what EFPIA hopes to achieve through participation in the NMP. 10 mins The floor will be opened for Q&A and discussion with the panelists. 20 mins Closing remarks and final comments by the panel. 5 mins
Moderators
Tarang Sharma, MS, MPH, PhD
WHO Regional Office for Europe, Copenhagen, 84, Denmark
Tarang Sharma has 20+ years of post-graduate experience as a biologist, epidemiologist and health economist. She has worked with regulatory submissions, health technology assessments and clinical guidelines, as an author, methodologist, reviewer, editor and decision-maker. Tarang Sharma has until recently worked for the Agenda, Policy & Strategy unit in the Immunization, Vaccines and Biologicals department at the World Health Organization HQ in Switzerland, supporting evidence to policy recommendations for new COVID-19 vaccines as well as cross-cutting considerations by the Strategic Advisory Group of Experts on Immunization (SAGE) and has previously worked for evidence to policy impact and health research mechanisms both at WHO HQ and their WHO European Regional Office (WHO Europe) in Denmark as a senior consultant and a technical officer. She now acts as the HTA focal point and is the Technical Officer for the Novel Medicines Platform at WHO Europe.
She has also previously worked for three different national governments: in USA (American Museum of Natural History) and in Europe for two HTA agencies: the National Institute for Health and Care Excellence [NICE] as a Senior Analyst in the United Kingdom and for the Danish Medicines Council: Medicinrådet, as a Specialist Consultant in Denmark. In UK, initially as a health economist at the University of Sheffield that hosts the NICE Decision Support Unit apart from undertaking individual HTAs for NICE, she was responsible for setting methods and standards for HTA. Then later, directly for NICE initially as a technical lead for the guidelines and then later with the science, policy, and research unit, as a senior analyst she managed a technical scientific team, to develop and improve methods, research, and partnerships for HTA. She also represented NICE at the international collaborations many funded by EU such as EUnetHTA, DECIDE apart from GRADE, social values in HTA etc. At Medicinrådet she helped establish the methods and processes for the use of quality adjusted life years approach as well as priority setting for HTA assessments, apart from conducting individual HTAs for Denmark.
She has also worked for non-governmental organizations (NGOs) with the Nordic Cochrane Centre (now referred to as Cochrane Denmark) in Denmark as well as part of the Cochrane Central Executive Team at their headquarters in London, UK. As a Research fellow, with Cochrane Denmark, she worked to improve transparency of clinical trials of medicines and reporting of adverse events and quality of life outcomes in collaboration with regulators EMA and the UK’s MHRA, for which she received a PhD from the University of Copenhagen. Later at the Cochrane headquarters, she established a new workstream to manage the editorial policies, process, budget and team for the Cochrane’s Fast-Track service (now the centralised service). She has also worked at the grass-roots level for NGOs in India for Akanksha and Kutumb foundations for nutrition and public health for populations living on the streets.
She has also worked for two pharmaceutical companies Novo Nordisk A/S in Denmark and Ranbaxy Laboratories Ltd (later bought by Sun Pharma) in India. At Novo Nordisk, she assessed quality of life and other patient reported outcomes from Phase III trials and conducted relevant observational studies for new medicines for submissions to authorities including discrete choice experiments, willingness to pay assessments, patient and carer surveys etc. As a Research Scientist at Ranbaxy, she conducted pre-clinical and Phase I trials for new medicines for their development as well as for generics.
She is an honorary associate editor for the Frontiers in Public Health: Health Economics and has previously been an editor for BMC journal Cost-effectiveness and Resource Allocation, BMJ, Cochrane and a series editor for the WHO Europe publication Health Evidence Network (HEN). She has a MS in biology (applied recombinant DNA technology, Jan 2003) from New York University, an MPH (specialised in health economics, November 2008) from University of Sheffield and MPH (July 2008) and a PhD in public health and epidemiology from University of Copenhagen (May 2018).
Panelists
Thomas Allvin, MA
European Federation of Pharmaceutical Industries and Associations, BRUSSELS, VBR, Belgium
Thomas Allvin is Executive Director Strategy and Healthcare Systems at EFPIA. Before joining EFPIA in 2015, Thomas worked for the Swedish Ministry of Health, including as health attache at the Permanent Representation to the EU.
Simone Boselli, MSc
Eurordis, Rare Diseases Europe, Brussels, Belgium
Simone Boselli joined EURORDIS in 2017 as public affairs director. A member of the European and international advocacy team, he contributes to policy development and represents EURORDIS in discussions on access to therapies, with a focus on reducing delays and inequalities, on the underlying challenges in the field of value assessment, pricing and reimbursement of orphan medicines, and on initiatives towards improved access. Mr Boselli contributed to EURORDIS’s position paper on “Breaking the Access Deadlock to Leave No One Behind”, and he supports advocacy activities at Rare Diseases International and the development of the NGO Committee for Rare Diseases.
Meindert Boysen, PharmD, MSc
Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Meindert is responsible at NICE for international partnership working, developing NICE’s role in international fora and representing NICE at key international meetings, further sustaining and developing NICE’s international reputation. Until recently, Meindert was the director at NICE responsible for delivery of the programmes that together form the centre for health technology evaluation. Including the technology appraisals, highly specialised technologies, medical technologies evaluation, diagnostic assessment and interventional procedures programmes. After qualifying as a pharmacist from Utrecht University, he worked in a hospital in the Netherlands, and held positions in health outcomes and sales in the pharmaceutical industry. Meindert completed the MSc in Health Policy Planning and Financing at the London School of Hygiene and Tropical Medicine and the London School of Economics & Political Sciences and briefly worked for the King’s Fund before starting at NICE in 2004. Meindert was a member of the Board of NICE and a director of the board of the International Society for Pharmacoeconomics and Outcomes Research (2017-2020).
Multi-Stakeholder Dialogue and Scientific Advice-Consultation in the New HTA Regulation
Digital Conference Pass
Level: Intermediate
ISSUE:
Data requirements for regulatory and health technology assessment (HTA) decision-making largely overlap. However, the difference in remits and scope alter the context of their decisions. This may lead to differences in uncertainty acceptance, which is critical from a global development standpoint when managing the various evidence expectations. Ultimately, this may diverge decision outcomes, reduce trust, delay or reduce patient access, and duplicate efforts for stakeholders. Diverging evidence expectations are profound in pharmaceuticals with high uncertainty, such as for rare diseases or paradigm shifting products such as tumor agnostics. The differing uncertainty acceptance can be seen in trust in biomarkers, surrogate outcomes, or trial designs. The HTAR has the potential to change the approach to evidence generation. It may allow for implementing new pathways across regulators and HTA (involving patients and clinicians) in the form of joint or parallel scientific consultations between the EMA and HTA. In one view, multi-stakeholder dialogues represent a solution. Discussing anticipated uncertainties, evidentiary requirements including post-launch and real-world evidence, or other risk management strategies, may result in aligned advice for the developer. Opponents might argue that variability is important in exposing differences in perspectives and that designing evidence programs fitting all stakeholders is a complex exercise. Additionally, decision-making and consensus-finding on a large scale is difficult due to the many interests. This issue panel will debate on whether evidence generation can benefit from an aligned multistakeholder advice or whether the variability of perspectives would make it unfit.
OVERVIEW:
The moderator will first present some recent findings related to decision-making under uncertainty. The panelists will reflect on which way the HTAR implementation can improve evidence generation. This will include the regulatory, HTA and developer perspective. Moderator: 10 min Panelist 1: 10 min Panelist 2: 10 min Panelist 3: 10 min Audience discussion: 20 min
Moderators
Milou Hogervorst, PharmD
Utrecht University, Utrecht, UT, Netherlands
PhD candidate at Utrecht university and market access manager at Roche NL.
Panelists
Wim Goettsch, PhD
National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He has serves as a special HTA advisor to Zorginstituut Nederland and a former director of EUnetHTA JA3 Directorate.
Inka Heikkinen, MSc, MBA
MSD, Copenhagen, 84, Denmark
Lead of the EFPIA Joint Scientific Consultation workstream. Special focus on regulator-EMA interaction, participated in various discussions and research activities (eg. on unmet medical need). Active in EHDS and RWE discussions and advocacy within EFPIA. Academic degrees on health economics and in policy, an Executive MBA degree with specialisation in finance.
Steffen Thirstrup, MD, PhD, CMO
European Medicines Agency, Amsterdam, Netherlands
Steffen Thirstrup is a medical doctor and board-certified specialist in clinical pharmacology and therapeutics. He holds a PhD in pharmacology and has a long background in clinical internal medicine with special emphasis on adult respiratory medicine. Additionally, Dr. Thirstrup was appointed adjunct professor in pharmacotherapy at the Faculty of Health Sciences, University of Copenhagen, in 2012.
From 2004-09 Steffen Thirstrup worked at Danish Medicines Agency first as the Danish member of CHMP at the European Medicines Agency (EMA) for five years including 10 months as joint CHMP- and CAT-member, followed by a short period as head of Danish Institute for Rational Pharmacotherapy dealing with HTA and best practice guidelines for primary care. In 2011 Prof. Thirstrup rejoined the licensing division at the Danish Medicines Agency acting as Head of Division for Medicines
Assessment and Clinical Trials. During this period Prof Thirstrup co-chaired the European Commission’s working group on market access for biosimilars medicinal products and acted as key scientific contact for the managing entity of the IMI beneficiaries for the PROTECT collaboration (Pharmacoepidemiological Research on Outcomes of Therapeutics by a European ConsorTium).
In March 2013, Prof Thirstrup joined the pharmaceutical consultancy company NDA Group AB as a full- time medical advisor on NDA’s regulatory advisory board. In April 2014 Prof Thirstrup was appointed as director for the Regulatory Advisory Board at NDA Regulatory Services Ltd.
Since June 2022 Prof Thirstrup has been the Chief Medical Officer at the European Medicines Agency, Amsterdam, The Netherlands
Podium Sessions
The Impact of Cancer Screening Programs on Population Health Outcomes
Digital Conference Pass
Moderator
Shilpi Swami, M.A. Economics
ConnectHEOR, London, LON, United Kingdom
Shilpi Swami, a seasoned Health Economics and Outcomes Research (HEOR) expert, currently serves as Vice President, HTA and Strategy at ConnectHEOR Consulting Company. She is also the Member Engagement Co-Chair at ISPOR Oncology Special Interest Group.
With a comprehensive global track record in leading HTA submissions and market access strategies, Shilpi's diverse experience spans academia, consulting, and biopharma, allowing her to offer strategic insights from multiple perspectives.
Formerly a Research Fellow at the University of York, Shilpi has contributed significantly to public health projects and academic best practices in health economics. She remains dedicated to improving healthcare through data-driven insights and evidence-based research.
P10: Impact of the COVID-19 Pandemic on Cancer Screenings in Portugal and Years of Life Lost Due to Delayed Diagnoses
2:00PM - 2:15PM
Mendes D 1 , Figueiredo D2 , Alves C3 , Penedones A3 , Costa B4 , Batel Marques F3 1 University of Coimbra, Porto Salvo, 11, Portugal, 2 University of Aveiro, Aveiro, Portugal, 3 University of Coimbra, Coimbra, Portugal, 4 Clevidence, Porto Salvo, Lisboa, Portugal
OBJECTIVES: This study aimed to evaluate the impact of the COVID-19 pandemic on cancer screening programs carried out by the Portuguese National Health Service (NHS), and to estimate the consequences on cancer morbidity and mortality.
METHODS: T
he pre-pandemic and pandemic periods were compared using publicly available data on performance and health outcomes indicators of the Portuguese NHS, namely the numbers and proportions of eligible individuals who underwent cancer screening (breast, cervical or colorectal). Pre-pandemic data were modelled to project hypothetical scenarios without a pandemic using an exponential smoothing algorithm, and then compared with data collected during the COVID-19 pandemic. A Markov model was developed to estimate years of life lost (YLL) due the reduction in the number of cancer screenings during the pandemic. The MS Excel and the PRISM symbolic model checker software were used.
RESULTS: There was
a decrease in the number of breast cancer (13%), cervical cancer (15%) and colorectal cancer (9% to 11%) screenings during the first two years of the pandemic. Average losses of 11 months, 6 months, and 4 months of life per patient with breast, colorectal and cervical cancer, respectively, were estimated. Additional 695 deaths and 3789 patients undiagnosed cancers were estimated in a 25-year time horizon, in comparison to the number predicted in a scenario without the pandemic.
CONCLUSIONS: This study quantified the impact of the COVID-19 pandemic on the Portuguese NHS activity. The disruption in the cancer screening programs
may contribute to increase cancer morbidity and mortality, with significant loss of years of life. T he long-term implications of the impaired cancer screening should be assessed, and proactive measures put in place to restore cancer screening levels and mitigate cancer morbidity and mortality indirectly attributable to the COVID-19 pandemic.
P12: Screen-Detected Breast Cancers Have Improved 5-Year Disease Free Interval Compared to Interval and Non-Screen-Related Breast Cancer, Taking Lead Time into Account
2:30PM - 2:45PM
Eijkelboom A 1 , de Munck L1 , Otten JDM2 , Broeders MJM2 , Siesling S3 1 Netherlands Comprehensive Cancer Organisation (IKNL), Utrecht, Netherlands, 2 Radboud University Medical Center, Nijmegen, Netherlands, 3 University of Twente, Enschede, OV, Netherlands
OBJECTIVES: We investigated the association between method of breast cancer detection and disease free interval (DFI), corrected for lead-time.
METHODS: Women, 50-76 years, diagnosed with breast cancer between 2005-2008 were selected from the Netherlands Cancer Registry. Data of women diagnosed between 2006-2008 were previously linked to the Netherlands Breast Cancer Screening Program. Three groups were defined: screen-detected cancer (SDC, diagnosed <24 months after a positive screening), interval cancers (IC, diagnosed <24 months after negative result), and non-screened cancers (NSC, all other cancers). Women diagnosed in 2005 were not linked and defined as SDC versus non-SDC. Data on recurrent disease (locoregional recurrences, distant metastasis, contralateral breast cancer) were obtained from patient files 5-years (2006-2008-cohort) or 10-years (2005-cohort) after diagnosis. Duffy’s method was used to calculate lead-time corrected follow-up, by subtracting each individuals lead-time from their observed follow-up, resulting in a maximum adjusted follow-up for SDC of 2.0 years (2006-2008-cohort) and 6.1 years (2005-cohort). Primary outcome was adjusted DFI, defined as being free from recurrent disease.
RESULTS: In 2006-2008, 15,176 women were diagnosed. After 2.0 years of lead-time corrected follow-up, DFI was 94.3% for women with a SDC, versus 93.6% of women with an IC, and 92.8% of women with a NSC. Women with an IC or a NSC had an increased risk of recurrent disease compared to SDC (HR: 1.16, 95%CI: 1.00-1.33, HR: 1.32, 95%CI: 1.13-1.54, respectively). In 2005, 6,215 women were diagnosed. After 6.1 years of lead-time corrected follow-up, DFI was 84.7% of women with a SDC, versus 80.6% of women with a non-SDC. Women with a non-SDC had an increased risk of recurrent disease, compared to women with SDC (HR: 1.30, 95%CI: 1.15-1.47).
CONCLUSIONS: Women with SDC have an improved DFI compared to women with an IC or NSC, which still remains after correction for lead-time.
P9: Impact of Organized Colorectal Cancer Screening on Age-Specific Population Incidences: Evidence from a Natural Experiment in Sweden
1:45PM - 2:00PM
Chauca Strand G 1 , Strömberg U1 , Forsberg A2 , Bonander C1 1 University of Gothenburg, Gothenburg, Västra Götalands Län, Sweden, 2 Karolinska Institutet, Stockholm, Stockholms Län, Sweden
OBJECTIVES:
Colorectal cancer (CRC) incurs a significant burden of disease globally. Organized CRC screening programs have been widely implemented for early detection and prevention. To understand the public health impact of these programs, quantitative evidence on changes in age-specific population incidences is fundamental. We aimed to provide such evidence by exploiting a natural experiment in Sweden, where two out of the 21 regions (comprising nearly 20% of the Swedish population) have offered organized screening since 2008.
METHODS:
Using registry data on diagnosed CRC cases and socio-demographics for all regions in Sweden over the period 1970–2021, Bayesian structural time series modeling and difference-in-differences were applied to analyze the impact of screening on age-specific population incidences over time (CRC cases per 100.000 persons/year).
RESULTS:
After successively inviting birth-year cohorts aged 60-69 years for stool-based testing, the incidence rate in the 70–74-year age group decreased significantly over time, with an average reduction of -44·40 (95% CI -58·15 to -31·31) from 2011 to 2019. In the overall population aged 60–74 years, there was a net incidence decrease of -7·99 (95% CI -13·85 to -2·39) since the initiation of organized screening in the intervention regions, 2008–2019.
CONCLUSIONS:
Organized CRC screening for 60–69-year-olds generated a change in age-specific incidence patterns with a long-lasting incidence decrease in the 70–74-year-old population, implying a reduction in the excess mortality and burden of the disease.
P11: Real-World Evidence of Total Colonoscopy for Colorectal Cancer Screening Based on Test Performance RCTs
2:15PM - 2:30PM
Hamashima C 1 , Terasaw T2 , Hosono S3 , Katayama T4 , Sasaki S5 , Abe K1 , Hoshi K6 , Tadano T7 1 Teikyo University, Itabashi, Tokyo, Japan, 2 Fujita Health University, Toyoake, Aichi, Japan, 3 National Cancer Center, Chuo, Tokyo, Japan, 4 Hyogo Prefecture University, Akashi, Hyogo, Japan, 5 St. Luke’s International Hospital, Tokyo, Japan, 6 National Institute of Public Health, Saitama, Japan, 7 Miyagi Cancer Association, Sendai, Miyagi, Japan
OBJECTIVES: Total colonoscopy (TCS) has been anticipated as a new method for colorectal cancer screening. Although randomized control studies (RCTs) are ongoing, there has yet to be a conclusive result. We evaluated the effectiveness of TCS screening following the stepwise approach developed by the World Endoscopic Evaluation (WEO).
METHODS: The WEO approach is to assess test accuracy and test performance for each new technique. We performed a systematic review of select studies during each phase by using of MEDLINE and Cochrane Library. We compared test accuracy for detecting advanced neoplasia (AN)/colorectal cancer (CRC) between fecal immunochemical testing (FIT), flexible sigmoidoscopy (FS), and TCS. Test performance indicators were compared based on single-round RCTs and performed a meta-analysis based on intention-to-treat (ITT) and per-protocol analysis.
RESULTS: One study compared sensitivities between FIT, FS, and TCS which were simultaneously performed. The sensitivity for the AN detection was consistently higher in TCS than others, even if the AN size was changed. 14 RCTs of test performance as colorectal cancer screening were selected. In the ITT analysis, the TCS participation rate of the meta-analysis was lower than FIT (RR= 0.49, 95%CI:0.22-0.89) and FS (RR= 0.83, 95%CI: 0.78–0.88). Although the AN detection rate was higher than FIT (RR= 2.25, 95%CI:1.40-3.61) and FS (RR= 1.15, 95%CI: 0.88–1.51), CRC detection was higher but not statistically significant (FIT RR= 1.48, 95%CI:0.66-3.43; FS RR= 1.08 95%CI: 0.49–2.37). On the per-protocol analysis, AN and CRC detection rates were higher in TCS than others.
CONCLUSIONS: Although high sensitivity was confirmed in TCS screening, test performance as a screening program was insufficient because of the low participant rates. One RCT presented that the efficacy of TCS screening could not be obtained for the same reason. The test performance of RCT could predict the effectiveness of the screening program.
Using Discrete Choice Experiments to Elicit Carers' and Patients' Preferences
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This podium explores how discrete choice experiments can be used to elicit preferences for patients' and carers' providers for various treatment options. Presentations will explore stroke patients' acceptance for digital technologies in neurorehabilitation, physicians’ preferences guiding the allocation of deceased donor organs, Parkinson's disease patients' preferences for remote monitoring, and carers' preferences for home care service when they provide care for Alzheimer's disease patients.
Moderator
Bill Byrom, B.Sc., Ph.D.
Signant Health, Nottingham, NTT, United Kingdom
Bill serves as Vice President at Signant Health, a global company providing technology solutions for pharmaceutical clinical trials. He has worked in the Pharmaceutical industry for 30+ years and is a recognised industry leader in eClinical product strategy, electronic clinical outcome assessments (eCOA), and decentralized clinical trials. Bill is an experienced scientific expert and the author of over 70 publications, and two industry textbooks on electronic patient-reported outcomes (ePRO). His recent scientific work includes the use of wearable and sensor technology, and bring-your-own-device (BYOD) eCOA in clinical trials. Bill serves on two industry expert task forces within ISPOR, is a member of the scientific leadership board of the Digital Medicine Society (DiMe), and a core contributor to the US Critical Path Institute’s eCOA Consortium. Bill is a senior visiting professor within Nottingham Trent University's Medical Engineering Design Research Group.
P13: Carer Preferences and Willingness-to-Pay for Home Care Services for Older People With Dementia: A Discrete Choice Experiment in the Milan Metropolitan Area
1:45PM - 2:00PM
Meregaglia M 1 , Manfredi S1 , Perobelli E1 , Rotolo A1 , Donati E2 , Notarnicola E1 1 SDA Bocconi School of Management, Milano, MI, Italy, 2 Fondazione Ravasi Garzanti, Milano, MI, Italy
OBJECTIVES: Dementia is a major health and social care challenge in developed countries where most people are cared for in their own homes. This study aimed to elicit carer preferences and willingness-to-pay for alternative bundles of home care services in the Milan metropolitan area.
METHODS: A binary discrete choice experiment (DCE) was administered to a sample of informal caregivers recruited through a network of non-profit organizations. The study received approval by the Ethics Committee of Bocconi University. An optimal orthogonal in the differences (OOD) design was used to create nine choice sets with Ngene. Four attributes articulated into three levels each were included based on literature review, expert opinion, and prior interviews with caregivers: 1) the number of home care hours per month; 2) the type of care (i.e., health, social, mixed); 3) the caregiver peer support group (i.e., none; among carers; with also professional support); 4) the monthly family’s cost (€). A mixed logit model was used to analyze the responses using Stata 17.
RESULTS: A total of 90 self-administered questionnaires were collected in January-April 2023. Two thirds of caregivers were female (66.7%) and children (65.6%), and mean age was 59.1±12.3 years. The mean age of people with dementia was 82.1±6.57; around two thirds (65.9%) were female and almost half (48.9%) had also a paid caregiver. The DCE results showed that increased home care hours, mixed health and social home care, caregiver meetings with professional support and lower monthly costs were mostly valued by carers. Some preference heterogeneity was detected in relation to socio-demographic characteristics; for example, female and older caregivers revealed a higher preference for receiving mixed and health care, respectively, for their care recipients.
CONCLUSIONS: The study results are expected to support policymakers in recalibrating public home care services for people with dementia and their informal caregivers according to social preferences.
P16: What Trade-Offs Are Women Willing to Make When Choosing Between Potential Treatments for Moderate-to-Severe Vasomotor Symptoms Due to Menopause?
2:30PM - 2:45PM
Heidenreich S 1 , Cutts K2 , Doldos A3 , Todorova L4 , Kim J5 , Hillard T6 , Simoncini T7 , Mikkola T8 1 Evidera, London, LON, UK, 2 Evidera, Bethesda, MD, USA, 3 Evidera, London, Greater London, UK, 4 Astellas Pharma Europe, Addlestone, Kent, UK, 5 Astellas Pharma Inc, Northbrook, IL, USA, 6 University Hospitals Dorset, Poole, Dorset, UK, 7 University of Pisa, Pisa, Italy, 8 HUS Helsinki University Hospital, Helsinki, Finland
OBJECTIVES: To elicit women’s preferences for treatments of vasomotor symptoms (VMS) due to menopause.
METHODS: Published literature, workshops with menopausal women and physicians, clinical data and qualitative interviews informed the design of a discrete choice experiment (DCE). In the DCE, women made choices between two hypothetical treatments and an opt-out that differed in frequency of moderate-to-severe VMS episodes, improvements in other menopause symptoms, time to symptom improvement (i.e., onset time), and 5-year risks of breast cancer, blood clots and bone fractures. The opt-out was specific to each woman. Data were analyzed using mixed logit (MXL) and latent class logit (LCL) models. To participate, women had to be 40 to 65 years old and experience at least 14 moderate-to-severe VMS episodes per week.
RESULTS: This analysis included a set of first recruited 537 women, from Australia (16.6%), Canada (13.8%), United Kingdom (18.6%), Germany (17.9%), Denmark (6.5%), Spain (23.7%) and France (19.7%). Women were on average 54.6 years old, employed (62.8%) and often experienced trouble sleeping (52.1%). Few women were using hormonal therapy (12.7%). On average, women accepted an extra 0.1% risk of breast cancer and blood clots for every 10.0% reduction in VMS frequency. Furthermore, they accepted an extra 1.2% risk of breast cancer and an additional 1.1% risk of blood clots for improvements in other menopause symptoms. They also valued reductions in bone fracture risks, but onset time did not affect preferences. Four preference groups were identified: Group 1 (25%) focused on VMS frequency, group 2 (11%) focused on other menopause symptoms, group 3 (33%) focused on treatment risks, and group 4 (31%) weighted all benefits and risks without an attribute of outstanding importance.
CONCLUSIONS: While women made benefit-risk trade-offs their treatment preferences were heterogenous. This suggests that treatment selection should consider individual priorities and concerns about long-term treatment risks.
P14: Understanding Patients' Preferences for Remote Monitoring in Parkinson's Disease: Insights from a Mixed-Methods Study
2:00PM - 2:15PM
Godoy C 1 , Miele F2 , Mäkitie L3 , Fiorenzato E2 , Bakker LJ4 , Uyl-De Groot C1 , Redekop K4 , van Deen W4 1 Erasmus University Rotterdam, Rotterdam, ZH, Netherlands, 2 University of Padova, Padova, Italy, 3 University of Helsinki, Helsinki, Finland, 4 Erasmus University Rotterdam, Rotterdam, Zuid Holland, Netherlands
OBJECTIVES: Remote patient monitoring (RPM) holds significant promise in Parkinson’s disease (PD) management. However, our understanding of the preferences of PD patients for RPM programs remains limited. This study aimed to bridge this gap by eliciting and examining PD patients' preferences for RPM through interviews and a discrete choice experiment (DCE).
METHODS: A mixed-methods approach was employed to elicit PD patients’ preferences in Finland and Italy. Initially, seven individual semi-structured interviews and five focus groups were conducted, comprising a total of 27 patients. Discussions revolved around three topics: disease detection, current monitoring process, and perceptions about RPM. Building on the insights gained from interviews and focus groups, a 12 choice tasks DCE survey was constructed. The survey also included questions pertaining to patient demographics, prior experience with wearables, and digital literacy. Preferences were estimated using a conditional logit model.
RESULTS: Patient concerns regarding data privacy, stigmatization, costs, and device intrusiveness emerged during the discussions. However, potential benefits of symptom control and disease progression management appeared to outweigh most technology-related inconveniences. A DCE was built incorporating five key attributes: effectiveness, monitoring intensity, time commitment for online questionnaires, video recording at home, and out-of-pocket costs for devices purchasing. Initial results (n=7) revealed a preference among participants for more effective RPM interventions (β = 1.15, P=.193) without video recordings (β = 0.90, P=.011). Notably, costs emerged as a significant factor in the preliminary analysis (β = -2.12, P =.002), despite its lesser emphasis in the qualitative analysis.
CONCLUSIONS: Qualitative findings revealed that patients expressed willingness to embrace RPM if it proves to be effective for symptom and disease progression management. However, initial DCE results indicate that while effectiveness and privacy remain important, the significance of costs is higher than anticipated. These findings are preliminary and require validation through a larger sample size in the ongoing study.
P15: Unraveling Acceptance of Healthcare Innovations in Neurorehabilitation: A Systematic Approach Through Health Preference Research
2:15PM - 2:30PM
Fischer AK
OBJECTIVES: Strokes present a challenge to the healthcare system, as the absolute number of strokes has increased despite a decline in stroke-related mortality. The demand for neurorehabilitation is growing, necessitating innovative solutions due to a shortage of professionals. The utilization of digital technologies offers the potential for increased efficiency and effectiveness. However, patient acceptance plays a crucial role in the successful adoption. Therefore, this study aimed to systematically analyze patients acceptance for digital technologies in neurorehabilitation.
METHODS: A discrete choice experiment (DCE) was conducted, involving a total of 1259 respondents. The DCE encompassed five technical aspects: explanation and presentation of therapy exercise, information in therapy, contact to healthcare professionals, patients’ choice in therapy process, and data processing. Additionally , therapy success within 6 months and a cost-attribute copayment per month were added. A fractional-factorial efficient Bayesian design (D-error) was used. The collected data were analyzed using a mixed logit model, and willingness-to-pay and uptake probabilities were calculated.
RESULTS: The analysis revealed that therapy success within 6 months was the most influential criterion, followed by the monthly copayment cost. However, the results also indicated that technical aspects, in addition to clinical and cost factors, significantly influenced the probability of uptake. Three alternative digital technologies were examined, assuming constant therapy success: a prototype of a humanoid robot, further development of a robot, and a digital application. Uptake probabilities were calculated based on willingness-to-pay results, and significant differences in uptake probabilities were observed among the alternatives (44% vs. 65% vs. 84%).
CONCLUSIONS: This study demonstrates a systematic approach to understanding the acceptance of healthcare interventions, particularly in the context of digital technologies for neurorehabilitation. The findings highlight the significant impact of changes in technical characteristics on the acceptance of these technologies. By incorporating this information into healthcare decision-making processes, the development, implementation, and patient engagement can be enhanced .
Breakouts: IP, WS, & OBS
Artificial Intelligence to Support HTA and Conducting HTA for Artificial Intelligence Technologies: Recent Developments and Reflections
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Level: Intermediate
PURPOSE: This workshop will present findings from the Next Generation Health Technology Assessment project (HTx) relating to the juncture between HTA and artificial intelligence (AI). It will explore both how novel AI methods may be used to enhance the conduct of HTA and important considerations for HTA organizations when assessing AI-based healthcare technologies. Attendees will learn about recent developments in both directions.
DESCRIPTION: There is increasing interest in how AI can be used to conduct HTA. It may have the potential to accelerate HTA processes, for example by automating evidence identification, or to directly inform reimbursement decision making, by facilitating a deeper use of real-world data and more personalised clinical and cost-effectiveness conclusions. Furthermore, the growing presence of AI-driven health technologies means HTA organisations will increasingly have to assess the value of interventions that use AI, which may introduce novel challenges.
Jamie Elvidge will provide a summary of the HTx project, and describe work to develop an AI extension to the CHEERS checklist (“CHEERS-AI”) for reporting economic evaluations of AI-based health technologies (12`). Gunjan Chandra will present an application of “explainable” AI, using real-world data to predict individual glycated haemoglobin responses to a range of possible treatments for type 2 diabetes, incorporating both clinical and socioeconomic predictors (12`). Antal Zemplenyi will summarise the recommendations from a multi-stakeholder workshop to support healthcare decision-makers in integrating AI into HTA processes, with a special focus on Central and Eastern European countries (12`). François Houÿez will discuss the patient perspective on how AI could contribute to develop more targeted health interventions and assist healthcare decision making that accounts for patients’ preferences, and what should done to build trust in AI-based interventions (12`). There will be time for audience interaction. This workshop will be of interest to HTA stakeholders, and developers and users of AI health technologies.
Discussion Leaders
Jamie Elvidge, MSc
National Institute for Health and Care Excellence, Manchester, United Kingdom
Jamie Elvidge, MSc is a health economist and scientific adviser in the Science Evidence & Analytics Directorate at NICE, UK. As part of the NICE Research team, he collaborates on a range of HEOR research projects. This includes HTx, a Horizon 2020 (EU) project to support patient-centred, societally oriented, real-time HTA decision-making.
Jamie has worked at NICE for 5 years, including as an economic modeller (Clinical Guidelines) and adviser (Technology Appraisals). He has over 12 years' experience in HEOR, including consultancy and academia. He holds an MSc in health economics (University of York).
Jamie's research interests include economic evaluation, HTA methodology, and environmental sustainability in HTA.
Discussants
Gunjan Chandra, MSc
University of Oulu, Oulu, 14, Finland
Gunjan, holding a Master's in Computer Science and Engineering with a specialization in AI, is actively pursuing her PhD at the University of Oulu in Finland. Her proficiency lies in leveraging AI methodologies to predict health outcomes, notably in tailoring glycemic control and treatment projections for both type 1 and type 2 diabetes individuals. Proficient in Python, R, and Matlab, Gunjan not only handles sensitive healthcare data with finesse but also excels in data security and information quality. Her expertise extends to crafting personalized predictive models for orphan diseases such as Myelodysplastic syndromes and T-cell Prolymphocytic Leukaemia. Additionally, her team is dedicated to constructing causalML models for AML patients, alongside conducting comprehensive cost-effectiveness analyses focused on type 2 diabetes.
Julien Delaye, MS
Eurordis, ., Belgium
Julien Delaye joined EURORDIS in September 2019 as an intern and moved to the position of Public Affairs Assistant on the Rare 2030 project. He then became Public Health Policy Junior Manager and now holds the position of Patient Engagement Manager in HTA.
Julien is responsible for organizing the participation (awareness, training, monitoring) of people living with a rare disease and their representatives in the European Cooperation on HTA and in national HTA procedures, as well as participating in research activities in the HTA field where EURORDIS is invited to take a role.
Prior to EURORDIS, he worked as an event coordinator for European Calcified Tissue Society (ECTS), was a volunteer for Osteogenesis Imperfecta Federation Europe (OIFE) and was a marketing intern at Sanofi – Copenhagen.
Antal Zemplenyi, PhD
University of Pécs, Pécs, Hungary
Antal Zemplényi, PhD, is a health economist with more than 5 years of experience in academia specializing in HTA, health economics and outcome research and over 10 years of experience in the financial management of hospitals. He is the past president of the Hungarian Health Economics Association and the Hungary Chapter of ISPOR. He is the head of the Health Technology Assessment Centre at the University of Pécs and a senior health economist at the Syreon Research Institute. He is currently a Fulbright scholar based at the University of Colorado.
How Should Decision Makers Deal With Uncertainty in the Evidence to Support Surrogacy Relationships in Cost-Effectiveness Analysis?
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Level: Introductory
PURPOSE: Introduce the audience to the role of surrogate outcomes in cost-effectiveness analysis intended to inform HTA, with a specific focus on how uncertainty should be dealt with using illustrative cases studies
DESCRIPTION: With the increasing use of flexible and accelerated regulatory pathways, the number of drug approvals using surrogate endpoints is increasing. Cost-effectiveness analysis requires quantitative evidence to model how changes to the surrogate outcome translate into changes to longer-term outcomes usually of interest to HTA bodies. When high quality evidence to support these relationships is missing, this can introduce considerable uncertainty in the evidence which can complicate decision making.
This workshop will present the ongoing work of an international collaboration of HTA agencies who are developing best practice guidance for the use of surrogate outcomes in cost-effectiveness analysis intending to inform HTA. The discussion leader will present an overview of the working group’s rationale and the questions it is seeking to answer (10 minutes), which include the regulatory standards for validating surrogate endpoints, to what extent these standards meet requirements for using surrogate outcomes in economic modelling, and how surrogate outcomes should feature in the model conceptualisation stage of the cost-effectiveness model. The discussants will then each present and discuss the complications that arise for a decision maker when high quality evidence to support surrogate relationships is missing using case studies (15 minutes per case study) such as a rare disease with high unmet need but with little evidence on long-term outcomes, a chronic condition where the surrogate relationship is well understood, and a condition with mixed evidence for the surrogate-long term relationship. These will be used for eliciting responses from the audience on how they believe the uncertainty should be dealt with and whether the evidence supporting the surrogate relationship is acceptable to inform decision making or not.
Discussion Leaders
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Jacoline is the Programme Director - Medicines Evaluation at NICE. She is responsible for the delivery of medicines guidance produced by NICE’s Technology Appraisals and Highly Specialised Technologies work programmes.
Discussants
Saskia Knies, PhD
Zorginstituut Nederland, Diemen, NH, Netherlands
Saskia Knies, PhD is senior advisor health economics and personalised medicine at the National Health Care Institute in het Netherlands (Zorginstituut Nederland; ZIN). In addition, till recently she was the coordinator of the research network HTA, which is a collaboration between ZIN and Erasmus University Rotterdam and Utrecht University. She holds a MSc in Health Science Research – specialisation HTA from Maastricht University and a PhD in health sciences also from Maastricht University. Due to her experience as pharmacoeconomic assessor she has extensive experience in using economic evaluations for reimbursement decision making. Saskia’s research interest are focused on HTA methods development to support healthcare decision making especially concerning economic evaluations, transferability of (health economic) evidence and on international comparisons. Saskia is also involved in the H2020 funded research project HTx. Her main task in all her work is building bridges between research (especially methods development or policy research) and the use of these methods in supporting decision making. Saskia has multiple publications in the field of HTA and pharmacoeconomics. She has been actively involved in different ISPOR activities, among others faculty member of a short course on transferability, member of ISPOR Taskforce on Value of Information, member of the HTA Roundtable Europe, reviewer for Value in Health and presenter in several panels and workshops. Saskia is also a guest senior researcher at the Erasmus School of Health Policy & Management (ESHPM) at Erasmus University Rotterdam.
Daniel Ollendorf, PhD
Tufts Medical Center, Boston, MA, USA
Dan Ollendorf is Director of Value Measurement and Global Health Initiatives at the Center for the Evaluation of Value and Risk in Health (CEVR). His research interests include expanding the use of health technology assessment in low- and middle-income economies, as well as refinement of value assessment tools in the US. Prior to joining CEVR, Dr. Ollendorf was Chief Scientific Officer for the Institute for Clinical and Economic Review (ICER) for over 10 years, where he was responsible for scientific oversight of all clinical and economic aspects of the health technology assessment process. As of November 2023, Dr. Ollendorf has returned to ICER as Chief Scientific Officer and Director of International HTA Methods and Engagement.
Dr. Ollendorf holds a PhD in clinical epidemiology from the University of Amsterdam and an MPH from Boston University. He is the current Chair of the Health Technology Assessment International Global Policy Forum as well as a non-resident Fellow at the Center for Global Development, and served on the US Medicare Evidence Development and Coverage Advisory Committee (MEDCAC) from 2015-2019. Dr. Ollendorf has authored over 125 articles in peer-review journals and is co-author of The Right Price: A Value-Based Prescription for Drug Costs (Oxford University Press).
Exploring Patient Experience Data Dossiers: A Mechanism to Connect Decision-Makers With Patient-Centered Evidence
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Level: Introductory
PURPOSE: Patient experience data (PED) are broadly defined as “data... collected by any persons... to provide information about patients’ experiences with a disease or condition, including the experiences, perspectives, needs, and priorities of patients.” Evidence derived from PED is spread across the peer-reviewed literature, white papers, reports, and patient-group websites. This disorganization makes it challenging for medical product development stakeholders and other healthcare decision-makers to identify PED to inform decision-making. One solution of interest is the concept of disease-specific “PED Dossiers” and a corresponding PED Dossier Directory linking to those PED dossiers. Building on global value dossier frameworks, e.g., the AMCP Format and EUnetHTA Core Model, disease-specific dossiers would be developed by patient groups to communicate PED collated from a variety of sources. During this interactive session, we will invite members of the HEOR community to discuss the draft PED dossier format and opportunities for dossiers to inform decision-making. DESCRIPTION: The audience will be invited to participate throughout the discussion via polls, Q/A, and prepared group discussion questions about strategies for driving acceptability/adoption across settings; uses specific to HEOR, including their potential role in implementation of joint scientific consultations in Europe and Medicare drug negotiations in the United States; and stakeholder roles in the generation and use of a Dossier. To begin, Elisabeth Oehrlein will provide an overview of PED Dossiers, including the challenge they are intended to address, a draft template currently being pilot tested, and stakeholder perspectives collected via interviews. Omar Escontrías will present the patient community perspective gathered through a series of workshops with patient groups. Next, Hayley Chapman will discuss strategies for stakeholders to collaborate internationally on PED Dossier implementation. Finally, Eleanor Perfetto will discuss how PED Dossiers align with priorities identified by members of ISPOR’s Patient-Centered Special Interest Group and Patient Roundtable.
Discussion Leaders
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Discussants
Hayley Chapman, MHS
Patient Focused Medicines Development (PFMD), Oakville, ON, Canada
Hayley Chapman is a Senior Program Director with Patient Focused Medicines Development (PFMD). She has a Masters in Health Science from the University of Toronto and has worked in the healthcare sector for over 25 years focusing on the development and evaluation of partnerships between life science companies, patients, and patient organizations.
With PFMD, Hayley leads several key strategic initiatives including Patient Engagement Activities in the Post Launch Phase, Monitoring Patient Engagement to Learn Strategies, and Patient Engagement & Patient Experience Data Fusion. These projects include co-creation with global, multi-stakeholder working groups to ensure that what matters most and is important to patients, family members, and caregivers is incorporated into decision-making for product development and within the healthcare system.
Hayley not only has a professional interest in patient engagement but has also contributed to several provincial and national Advisory Boards sharing her caregiver perspective following her son's cancer diagnosis and treatment. She is currently a member of both the Oncology Family Advisory Council and the Patient and Family Advisory Council at Sick Children's Hospital in Toronto.
Omar Escontrias, DrPH, MPH
National Health Council, Washington, DC, USA
Dr. Omar A. Escontrías’ expertise is centered in the areas of community and patient engagement, coalition building, health policy, and evidence-based research. Over the span of his career, Dr. Escontrías has worked with local and state governments, as well as non-profit organizations in the areas of public policy, disease prevention, and health promotion to improve the health and lives of patients and communities. By the time he joined the National Health Council (NHC) in April 2022 as its Senior Vice President of Equity, Research and Programs, he had already spent 15 years building a career on fighting systemic health care inequalities in historically underrepresented communities. Dr. Escontrías is a proud first-generation immigrant and is thrilled to be contributing to the NHC’s patient-centered advocacy, health equity, and policy engagement efforts. Dr. Escontrías earned a Bachelor of Science in molecular and cellular biology, a Master of Public Health in epidemiology, and a Doctor of Public Health in health policy and management, all from the University of Arizona.
Eleanor Perfetto, PhD, RPh, MS
Pharmaceutical Health Services Research, School of Pharmacy, University of Maryland School of Pharmacy, VENICE, FL, USA
Eleanor M. Perfetto has worked in government, industry, consulting, and academia. Most recently, Eleanor was with the leading, U.S. nonprofit organization focused on patient issues as Executive Vice President for Strategic Initiatives at the National Health Council (NHC). Currently, she holds a faculty appointment at the University of Maryland School of Pharmacy where she is Professor of Pharmaceutical Health Service Research. Her research and policy work primarily focus on patient and family engagement, including comparative effectiveness and patient centered-outcomes research; patient-focused drug and medical-product development; clinical outcome assessment, selection, and development; and patient-centered value assessment and healthcare quality measurement.
As caregiver for her late spouse who suffered from early dementia due to chronic traumatic encephalopathy, Eleanor is a recognized advocate for patients with head-trauma-related dementia and their families. In May 2007, she received the Alzheimer’s Association New York City Chapter Advocacy Leadership Award. In 2010, she received the Alzheimer’s Association National Capital Area Chapter Award for Advocacy & Awareness. In 2012, she received the Legacy Award from the Concussion Legacy Foundation, for which she is a past board president and a two-term inaugural board member. Today, she serves on the board for the Brain Injury Association of America, where she chairs the Governance Committee and serves as Board Secretary. She also is a current board member for the Dan Lewis Foundation, which is dedicated to brain regeneration research.
Prior to joining the NHC and University faculty, Dr. Perfetto was with Pfizer Inc for almost eight years, most recently as Senior Director, Federal Government Relations; past positions were in Evidence-Based Strategies and Payment Policy Analysis. Early in her career, she served in the U.S. Public Health Service serving for 6 years as a pharmacist in the Indian Health Service in Pine Ridge, SD and Talihina, OK, and as senior pharmacoepidemiologist at the Agency for Health Care Policy & Research (now Agency for Healthcare Research & Quality). Her highest rank was Commander (05).
Dr. Perfetto has a distinguished record of service to professional and other organizations. Currently, she is Chair of the Patient-Centered Special Interest Group at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and served as co-chair for the 2016 ISPOR Annual Meeting. In the past, she has served as a CMS MedCAC member; co-chair of the National Quality Forum’s Alzheimer’s Disease and Related Dementias Project and on the Patient-Reported Outcomes Expert Panel; a past Pharmacy Quality Alliance and Drug Information Association board chair; and past member of the Center for Medical Technology Policy Board and Health Industry Council Board of Advisors. She was an Assistant Editor for the Journal of Managed Care and Specialty Pharmacy and has been a peer reviewer for numerous journals, the Patient-Centered Outcomes Research Institute, government granting agencies, and pharmacy professional organizations. She has contributed book chapters and published in peer-reviewed journals. She is a frequent speaker at numerous national and international scientific conferences.
Dr. Perfetto holds BS and MS degrees in pharmacy from the University of Rhode Island and in 2009 received the Distinguished Alumni award from the College of Pharmacy. She also holds a PhD in Public Health, concentrating in health policy and epidemiology, from the University of North Carolina at Chapel Hill.
ISPOR Student Network Session
A Strategic Symphony: Converging RWE, HTA, and Policy in Healthcare Decisions
The ISPOR Student Network Forum offers opportunities for student attendees to hear from experts on a wide array of subject manner ranging from career advice to scientific topics. In this in-person format, the event will welcome two presenters to each discuss their perspective on the topic at hand with attendees. The session will conclude with an open discussion and Q&A with the audience.
Moderators
Dominique Seo, MPH
University of Maryland, Baltimore, Baltimore, MD, USA
Speakers
Adam Brooke, MSc
NICE, Manchester, United Kingdom
Adam Brooke is an HTA Adviser for the NICE Technology Appraisals team. He has a Master’s in Epidemiology and Biostatistics from the University of Leeds. He has experience with a wide range of medicines appraisals including NICE’s appraisals of histology independent treatments and advised committees on treatments for COVID. He co-leads the NICE technology appraisals Pathway pilots, looking at creating and maintaining disease-specific reference models.
Eberechukwu Onukwugha, BA, MSc, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eberechukwu Onukwugha, PhD is a Professor in the Department of Practice, Sciences, and Health Outcomes Research and is the Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Master of Science in agricultural and applied economics as well as a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Tech. Dr. Onukwugha has approximately 20 years of experience conducting health economics and outcomes research. She examines the costs and health outcomes associated with health-related decisions as well as the institutional and environmental context framing these decisions.
Catia Proenca, PhD, PGDip
Alira Health, Basel, BS, Switzerland
Catia Proenca has over 11 years of experience in fundamental Research, RWE, and Market Access, across the pharmaceutical industry, consulting, and academia. Catia has expertise in designing and executing fit-for-purpose RWE strategies to meet key stakeholder needs (e.g., Payers/HTAs, KOLs, patients), closing evidence gaps to support the development of value propositions, and preparing a positive environment to enable access to technologies.
Catia holds a Ph.D. in Neuroscience from the Weill Cornell Graduate School of Medical Sciences, and a post-graduate diploma in epidemiology from the London School of Hygiene and Tropical Medicine. She has authored and co-authored several peered-reviewed publications. She has studied, worked and lived in Portugal, Germany, US and Switzerland.
Sunny Sheth, PharmD, MBA
Novo Nordisk, Livingston, NJ, USA
Recent PharmD & MBA grad now working in an academic research setting pursuing a Masters in Science in Health Outcomes, Policy & Economics. Contracted out to a pharmaceutical role in Novo Nordisk supporting the Evidence Synthesis & Value Assessment team 4 days a week ago first the first year of this two year fellowship.
14:45 - 15:15
Coffee Break
Located in the Exhibit Hall
14:45 - 15:45
Member Group Meetings
ISPOR Poland, Hungary, and Ukraine Joint Chapter Meeting
Come and connect with the ISPOR Hungary, Poland and Ukraine chapters during this joint meeting, open to all interested in learning about current trends in the Central and Eastern Europe region. The chapters will also share perspectives on priority topics in their respective countries. This is a great opportunity to network with colleagues and discover how to actively engage with these chapters.
15:00 - 16:00
Competency Framework Update
Professional Competencies for Health Economics and Outcomes Research – 2023 Update to the ISPOR Competencies Framework
In 2020, ISPOR’s Institutional Council, Educational Council, and Faculty Advisor Council published the Health Economics and Outcomes Research (HEOR) Competencies Framework™. Due to the evolving HEOR landscape, an ISPOR Competencies Framework Cross-Council Working Group was formed to update the professional competencies.
The goal of this forum is to present the work in progress of the update to the ISPOR Competencies Framework. The objectives are (1) for the panel to present an overview of the content of the update, which includes refining the existing Statistics and Analytics competency and adding new competencies related to diversity, equity, and inclusion (DEI), artificial intelligence/machine learning (AI/ML), and digital health, and (2) for the audience to provide input on the importance and relevance of the new competencies in small group discussions.
Ebere Onukwugha will open the session by welcoming attendees, introducing the speakers and highlighting forum objectives. Soham Shukla, Stephanie Earnshaw, and Laura Pizzi from the Cross-Council Working Group leadership will present on the history and process to update the competencies. Soham will offer an overview of the new competencies and lead the small group discussion, with support from the other speakers, offering perspectives on DEI, AI/ML, and digital health. Laura will close by sharing next steps and inviting participants to continue to give feedback.
Moderators
Eberechukwu Onukwugha, BA, MSc, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Eberechukwu Onukwugha, PhD is a Professor in the Department of Practice, Sciences, and Health Outcomes Research and is the Executive Director of Pharmaceutical Research Computing at the University of Maryland School of Pharmacy. She received a Master of Science in agricultural and applied economics as well as a Doctor of Philosophy in economics (concentration: econometrics) from Virginia Tech. Dr. Onukwugha has approximately 20 years of experience conducting health economics and outcomes research. She examines the costs and health outcomes associated with health-related decisions as well as the institutional and environmental context framing these decisions.
Speakers
Stephanie Earnshaw, PhD, MS
RTI Health Solutions, Pittsboro, NC, USA
Stephanie Earnshaw is senior vice president of Health Economics at RTI Health Solutions (RTI HS). She received her PhD in Industrial Engineering at North Carolina State University and has performed health outcomes and health services research for over 30 years, 24 of which have been with RTI-HS. She has presented workshops and various courses on decision-analytic modeling techniques for pharmaceutical companies and organizations such as ISPOR, the Academy of Managed Care Pharmacy (AMCP), and the Centers for Disease Control and Prevention (CDC). Dr. Earnshaw has served on the ISPOR Board of Directors and as Chair of the Audit Committee and is the current Educational Council Chair. She has held an Adjunct Faculty appointment at the University of North Carolina’s Eshelman School of Pharmacy, Division of Pharmaceutical Outcomes and Policy, is honored as a Distinguished Alumni in Industrial and Systems Engineering at North Carolina State University and is one of the lead authors of “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Dr. Earnshaw’s research focus is in applying decision-analysis techniques to industry-related issues and health care problems. Her areas of specialization include mathematical programming (constrained optimization), network optimization, and Markov, simulation, and other state transition modeling. In addition to developing budget-impact and cost-effectiveness models to support health technologies for the pharmaceutical, biotechnology, and diagnostic and medical device industry, she has developed innovative mathematical models using these methods to determine pricing strategy, predict clinical outcomes, allocate resources, and cost care pathways particularly in support of medical diagnostics. Therapeutic areas include cardiovascular disease, gastrointestinal disorders, respiratory disease, acute care, infectious disease, osteoporosis, vaccines, and oncology. She is a member of ISPOR and the Institute for Operations Research and the Management Sciences. She has presented her work at professional conferences and has published in several peer-reviewed journals.
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
Soham Shukla, PharmD
GlaxoSmithKlein, Paramus, NJ, USA
Soham Shukla, PharmD, MS is an associate director in the Value Evidence & Outcomes group at GSK, and resides in New Jersey, USA. He is the co-lead of the workgroup in charge of updating the ISPOR HEOR Competencies Framework. Soham trained as a pharmacist and has a degree in Health Outcomes, Policy, and Economics (HOPE), both from Rutgers University. Soham first joined ISPOR as a student and, through the years, has been an active member of the organization through the Rutgers student chapter, Health Equity SIG, Health Preference SIG, and serving as an Editorial Advisory Board Member of Value & Outcomes Spotlight.
15:15 - 15:45
Exhibit Hall Theater
How to Prepare for JCA in Local Health Systems? Ensuring HTA, Payer, and Industry Readiness to Accelerate Patient Access to Innovation
Digital Conference Pass
The countdown is on for the implementation of the EU Joint Clinical Assessment (JCA), but numerous questions linger especially for local country healthcare stakeholders.
This session is designed to provide a comprehensive roadmap based on current understanding of the challenges and the opportunities to successfully navigate the JCA process in European countries.
Our panel of experts, with knowledge from pilot assessments that already have been conducted, will delve into the intricate interplay between local Health Technology Assessment (HTA) bodies, payers, and the pharmaceutical industry, emphasizing the critical importance of collaboration and early engagement for successful JCA leverage.
The panel will cover essential aspects such as industry readiness, HTA body readiness and engagement, country payer engagement, regulatory considerations, and strategies for harmonious collaboration.
The ultimate goal of improving patient access to innovative treatments will be central throughout, reflecting the ethical and patient-focused dimensions of JCA process.
The presentation aims to equip attendees with actionable insights, fostering a proactive approach to JCA within local health systems and facilitating the acceleration of patient-centric innovation.
Sponsored by Cencora
Sponsor
Cencora
Moderators
Herbert Altmann, PhD
PharmaLex GmbH, part of Cencora, Munich, Germany
As Head Market Access, P&R Europe for Pharmalex/Cencora I´m managing since Feb 2023 the pan European Market Access activities. It is my objective to successfully engage with pharma companies, supporting them to bring innovative medicines to patients with unmet medical need as fast as possible, with a well-recognized value for the manufacturer.
After leaving Munich University with a PhD in Chemistry (thesis: Gentechnology), I worked for 3M Innovation and Amgen in different international/global leadership roles and the last 17 years at Novartis - last 10 years as Head of Value & Access Europe.
Speakers
Emer Fogarty, BSc (Pharm), MSc, PhD
National Centre for Pharmacoeconomics, Dublin, Ireland
Emer Fogarty is the Lead on HTA Methodology and Guidelines in the National Centre for Pharmacoeconomics (NCPE), Ireland. At the NCPE, Emer is responsible for the development of assessment methods, and is a Senior HTA Assessor. Emer currently represents Ireland on the HTA Coordination Group Joint Clinical Assessment Subgroup, and has previously worked as a clinical pharmacist, across numerous specialties including general medicine, oncology, and infectious diseases. Emer studied Pharmacy and received her PhD in Pharmacoeconomics from Trinity College Dublin, Ireland.
Matias Olsen, MSc
European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), Brussels, Belgium
Matias Olsen is the Senior Manager, Public Affairs and Policy for the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE). Matias supports EUCOPE on key topics, covering among others, implementation of EU HTA, advanced diagnostics, Real-World Evidence, the Patent Package, Europe’s Beating Cancer Plan and relevant market access topics. He coordinates members’ thematic working groups, including the P&R/Market Access Working Group, the Genomics Working Group and the EU HTA Regulation Task Force.
Matias has previously advocated on behalf of European cancer patients and worked in the Norwegian Social Security Administration, with reimbursement of medical devices and coordination of social security within the EU/EEA.
15:15 - 16:00
Student & Faculty Meet and Greet
Come meet the student and faculty leaders and converse with your peers about all things pertaining to ISPOR Student Chapters! Faculty will have the opportunity to meet with other members from the Faculty Advisor Council, and Student Leaders will also be able to meet with their peers and faculty.
15:15 - 16:15
Educational Symposia
Navigating the AI Revolution in Health Technology Assessments: Balancing Bias, Ethics, Time, Quality, and Evergreen Evidence
Digital Conference Pass
The rapidly evolving healthcare landscape, driven by growing pipelines, Big Data, targeted therapies, and breakthrough medicines, has paved the way for the transformative power of Artificial Intelligence (AI) in generating health economic evidence and conducting health technology assessments (HTA).
Throughout this educational symposium, attendees will have the opportunity to delve deep into the responsible deployment of AI, recognizing the importance of reducing bias and upholding ethical standards. The session will also shed light on the optimization of processes and implementation, and the maintenance of high-quality standards in decision-making.
We will explore the potential applications of AI, while acknowledging the crucial role of evergreen or living evidence in the evaluation of healthcare interventions. Evergreen evidence entails the continuous integration of new data, allowing for dynamic decision-making that reflects real-world healthcare outcomes over time – but can pose a challenge for traditional static evidence approaches.
Learn how to navigate the complexities of incorporating evergreen evidence into the AI-driven HTA landscape, ensuring that decision-making frameworks remain adaptable, relevant, and informed.
The goal of this multi-stakeholder panel is to parse out the noise and begin to drive a consensus around defining the boundaries for utilizing AI in healthcare decision-making. The panel will focus on the responsible deployment of AI, with emphasis on reducing bias, maintaining high ethical standards, and balancing the time needed for preparation while maintaining quality.
Sponsored by Parexel International
Sponsor
Parexel International
Moderators
Jackie Vanderpuye-Orgle, PhD
Parexel International, Boston, MA, USA
Jackie Vanderpuye-Orgle is a VP and the Global Head of Advanced Analytics at Parexel. She has extensive experience in providing technical expertise in data visualization, meta-analysis, AI, and statistical analysis. Focusing on underscoring the differentiated value of therapeutic assets, Jackie is adept at developing health economics and real-world evidence generation plans to facilitate market access. With over 20 years of experience as a health economist, Jackie has worked at Amgen, Precision Health Economics, and Analysis Group, among other organizations. She completed her PhD at Cornell University following a pre-doctoral program in economics at Yale University.
Speakers
Raquel Aguiar-Ibáñez, MSc
Merck Canada Inc, Kirkland, QC, Canada
Raquel is a senior director in oncology-related outcomes research at the Global MSD team. She currently leads the development of the evidence generation strategy and the execution of HEOR studies in oncology at a pan-tumor level. Raquel has extensive expertise in the development of global and country-specific pharmacoeconomic models, systematic reviews and HTA dossiers that have supported HTA submissions for multiple oncology indications across the world.
Raquel has a BSc in Economics from the University of La Laguna (Spain), and an MSc in Health Economics from the University of York (UK). Before joining MSD in 2014, Raquel held several HEOR-related positions, including: technical advisor in the Canaries Health Service (Spain); research fellow at the University of York (at the Centre for Reviews and Dissemination and at the York Health Economics Consortium, UK) and at the London School of Hygiene and Tropical Medicine (London, UK); senior project leader at i3Innovus; and senior health economist at Amaris Consulting.
Raquel is currently part of the R for HTA Scientific Committee, and was a member of the ISPOR Oncology Modeling Working Group in 2017. Between April 2016 and November 2017, Raquel was an industry member of the SMC User Group Forum (UGF), and between 2015 and 2016 she was a member of the Association of the British Pharmaceutical Industry (ABPI), contributing to the Pharmaceutical Oncology Initiative (POI) and the HTA Working Groups. She has authored and co-authored more than 25 peer-reviewed journal papers.
Rito Bergemann, MD, PhD
Access Consulting, Parexel International (CH) AG, Loerrach, Germany
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Associate Director (Research) at the National Institute for Health and Care Excellence (NICE). She has practised as health economist and researcher for over 20 years. Her current work is focused on advancing HTA and clinical guideline development methods through research.
Dalia leads NICE newly established HTA Innovation Laboratory (HTA Lab) as well as NICE’s portfolio of European Commission funded projects, such as IMI EHDEN and HORIZON 2020 HTx projects, with cumulative funding of over 5 Million Euros. She is widely published in the area of health economics and outcomes research and serves as Associate Editor of ISPOR journal Value in Health and as Director on ISPOR Board of Directors (2023-2026).
Denise Meade, MBA
Microsoft, Norwell, MA, USA
Sugandh Sharma, MSc
Parexel International, Mohali, Punjab, India
Sugandh is a Senior Director at Parexel with 17+ years of experience. She is an expert in HEOR & Market Access and has worked across consultancies successfully supporting clients achieve their business goals. Over the years, she has built deep expertise in conducting literature reviews and has been exploring the use of latest technologies to deliver faster insights. Keeping patients first, she is a passionate advocate for responsible adoption of innovations such as AI to ensure ethical and balanced progress.
How Can We Shape HTA with Real-World Evidence to Encourage Rare Disease Innovation?
Digital Conference Pass
Rare diseases pose unique challenges in health technology assessment (HTA), where stakeholders must make decisions in the face of greater uncertainty in the evidence base than is seen in more prevalent diseases. Health-care systems must address the large unmet need in rare disease patient populations while maintaining equity of access for them; however, challenges in terms of data, methodology and process implementation remain. Real-world data is a key source of evidence to reduce this uncertainty, complementing limited clinical trial data. Evidence derived from real-world data on natural history, comparative effects, resource use, quality of life etc. may be able to answer questions which are crucial for decision making. In recent years, certain HTA agencies have been active in trying to incorporate real-world evidence (RWE) and adjustments for rare diseases into their assessments. Advances have been made in methods and process guidelines, but have these led to meaningful changes in wider and faster access for patients? Are further theoretical advances required, or are there other, practical barriers to implementation? In which areas could RWE be transformative, and where will other solutions be required? How will the upcoming EU Joint Clinical Assessment process approach this issue? This symposium includes speakers with deep experience in a variety of aspects of HTA, who will discuss the current status of appraisal frameworks for rare disease treatments, how far RWE can go to resolve some of the uncertainties faced by HTA decision makers, and what the future may hold.
Sponsored by Biogen
Sponsor
Biogen
Moderators
Josie Godfrey, MA
JG Zebra Consulting, Hammersmith, United Kingdom
Josie has worked in rare diseases and innovative therapies for 14 years. She currently runs a consultancy business specialising in strategic market access, policy and stakeholder engagement. She is the Strategic Director for Duchenne UK’s Project HERCULES, an award-winning global collaboration developing evidence and tools to support HTAs for new treatments for Duchenne Muscular Dystrophy. Josie is also co-founder and joint CEO of Realise Advocacy, which supports patient involvement in drug development and access processes. She previously led work at NICE to establish the Highly Specialised Technologies programme.
Speakers
Anthony Hatswell, PhD
Delta Hat, Nottingham, DBY, United Kingdom
Anthony Hatswell is a statistician & health economist. After his education at the University of York he spent time in pharma before moving to consulting where he specializes in applied statistics. His current research looks at cross study comparisons, and analysis of utility data.
Pall Jonsson, BSc, PhD
Data and Analytics, National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Pall Jonsson is Programme Director for Data and Real World Evidence at the National Institute for Health and Care Excellence (NICE). His team has a strategic role in ensuring NICE is at the forefront of harnessing new and emerging opportunities for using real world data to inform NICE’s guidance to the health and care sectors.
Pall has a PhD in bioinformatics from the University College London. Before taking up his current role, he was Associate Director for Science Policy and Research, responsible for NICE’s portfolio of international research projects in areas such as big data and real-world evidence. Earlier experience includes work in academia, biotech and the pharmaceutical industry.
Mark Larkin, PhD, MA
Vitaccess Ltd, Oxford, United Kingdom
Mark Larkin is a real-world evidence expert who founded Vitaccess to improve understanding of patients' and caregivers' experience.
Vitaccess uniquely combines groundbreaking patient-centred research studies, powered by its proprietary Vitaccess Real platform. Primary data generation studies have been run in the US, Japan, Europe and Canada, with data available in close to real-time.
Elena Nicod, PhD
Dolon Ltd, London, United Kingdom
Elena is a Director at Dolon Ltd, a strategic pricing and market access consultancy specialized in rare and severe diseases. She is also co-lead of a Work Package in IMPACT-HTA that developed guidance on appraising rare disease treatments. Elena previously worked as a research officer at LSE Health, clinical trial unit coordinator at the University Hospital of Lausanne, and administrator of the Swiss Vaccine Research Institute. She completed a PhD in Social Policy at LSE, MSc in Health Policy Planning and Financing at LSHTM, and BSc in business administration at HEC Lausanne. She published more than 20 peer reviewed papers and is an expert in the OrphVAL and OD Expert Group initiatives.
Discussion Groups
Exploring Patient Experience Data Dossiers: A Mechanism to Connect Decision-Makers With Patient-Centered Evidence
Speaker
Elisabeth Oehrlein, PhD, MS
Applied Patient Experience, LLC, Washington, DC, USA
Navigating Challenges and Seizing Opportunities: Leveraging Multiple RWD Sources in External Control Arms for HTA and Regulatory Decision-Making
Moderator
Chris Cameron, MSc, PhD
EVERSANA, Sydney , NS, Canada
Dr. Chris Cameron is Chief Scientific Officer at EVERSANA. He is a global thought leader in health economics and outcomes research with over 15 years of experience. Prior to joining EVERSANA, Chris was a partner at Cornerstone Research Group Inc., and lead of Health Economics at CADTH. He has also been a consultant decision scientist for ICER, a member of the Economic Guidance Panel at pCODR, and served on PMPRB Technical Panel on drug pricing guidelines in Canada. Chris is an adjunct professor at Dalhousie University, and has authored over 180 peer-reviewed publications in many of the world’s top journals, including JAMA, Lancet, and BMJ. His research has been featured in the Globe and Mail and New York Times. He was recently named one of the most prolific authors in the world on indirect treatment comparisons and was a co-author on the PRISMA NMA reporting guidelines. Chris holds a bachelor’s degree in mathematics and an MSc and PhD in Epidemiology where he was a Vanier Canada Graduate Scholar. He also completed a fellowship on real-world data analytics at Harvard University and FDA’s Sentinel Initiative.
ISPOR Forums
An Effective Drug Is Not Enough: How Do We Improve Access to Molecular Testing to Enable Personalized Treatment in Oncology?
P recision oncology has allowed for more personalized treatment, often requiring molecular testing to identify those expected to show improved clinical bene fi t compared with other treatment options. However, access to testing can b e limited by a wide range of barriers . Despite efforts to address these barriers, challenges remain, and the consideration of multiple stakeholder perspectives will be necessary to identify effective solutions. I n this forum session led by ISPOR’s Oncology SIG , the panelists will discuss barriers to molecular testing access in cancer care from the patient, research, and industry perspectives. The goal of this forum is to better characterize this multifaceted issue, including current evidence gaps, and identify practical and impactful solutions that will be feasible from the HEOR perspective, optimizing patient outcomes while minimizing additional cost s. Dr. Urvalek will give a 10-minute presentation on industry - led initiatives and studies aimed at removing barriers to molecular testing and measuring impact . These collaborations involve community-based hospitals , diagnostic companies , and payors . Dr. Roydhouse will give a 10-minute presentation on research examining patient perceptions regarding the use of genetic information in cancer diagnosis and treatment. She will also present research and data collection considerations for understanding the impact and use of molecular testing outside of the clinical trial setting. Presentations will be followed by a moderated discussion and Q&A session with the audience. Outputs of the forum will inform future SIG activities , including a potential follow-up publication.
Moderators
Brittany Carson, PhD, MHEcon
ApotheCom, New York, NY, USA
Brittany is a Medical Director at a medical communications agency. She brings
extensive experience across multiple disease areas, from an in-depth mechanism of disease understanding to appreciation of clinical challenges and barriers to access to care. Her work has allowed her to explore communication to a wide variety of audiences in various formats, including publications, animations, and applications. She is also the Chair-Elect of the ISPOR Oncology Special Interest Group. Brittany received her PhD in pharmacology and has a master’s degree in health economics and holds authorship on numerous scientific publications.
Speakers
Jessica Roydhouse, PhD
Menzies Institute for Medical Research, Hobart, TAS, Australia
Dr Jessica Roydhouse is Select Foundation Senior Research Fellow in Health Services Research at the Menzies Institute for Medical Research, University of Tasmania, Australia. In this role she also serves as Director of the Tasmanian Cancer Registry. Her research interests include patient-reported outcomes, cancer clinical trials and missing data.
Alison Urvalek, PhD
Loxo@Lilly, Astoria, NY, USA
Alison Urvalek is currently an Executive Director in Clinical Diagnostics at Loxo@Lilly where she leads external research collaborations aimed at identifying approaches to increase comprehensive molecular testing for patients with cancer. Prior to this role she worked as a Diagnostic Medical Science Liaison focused on education around identifying appropriate patients who may benefit from precision medicines. Before joining Loxo@Lilly Alison worked in a medical communications and advertising agency supporting drug development and launch endeavors. She holds a PhD in Biomedical Science from Albany Medical College and completed a postdoctoral fellowship at Weill Cornell Medical College.
Artificial Intelligence-Enabled “Research Assistants” in Health Economics and Outcomes Research – Hire or Fire?
The new generation of large language models such as ChatGPT4 has heralded an era of highly efficient artificial intelligence (AI)-enabled “research assistants”. Should researchers in the health economics and outcomes research (HEOR) field be hiring these AI-enabled research assistants? Some said that the change has arrived and is irreversible. If so, what will be the most ethical, equitable and efficient use of these AI-enabled research assistants? Will these AI-enabled research assistants put us out of job?
This session will begin with a poll of the audience to understand the current practices with regards the use of AI-enabled research assistants. This is followed by a panel discussion with speakers from publishing, academia and the industry on their experiences with the use of AI-enabled research assistants in their routine work, current good practices, future trends and potential pitfalls to watch out for. The session will end with another poll of the audience to understand what guidelines for the field would they like to see.
Moderators
Hwee-Lin Wee, PhD
National University of Singapore, Singapore, Singapore
A/P WEE Hwee Lin is Associate Professor at the Saw Swee Hock School of Public Health at the National University of Singapore. She is a member of the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council, member of the International Editorial Advisory Board for Journal of Patient Reported Outcomes, and member of the National Advisory Committee on Cancer in Singapore. A/P Wee’s current work focuses on the use of real world evidence in health technology assessments.
Speakers
Yumi Asukai, MSc
Gilead Sciences, London, LON, United Kingdom
Yumi is a health economist specialising in economic modelling and evaluations for health technology appraisal. She received her BA in Political Science from Stanford University, before making the move to study health economics at the London School of Economics and the London School of Hygiene and Tropical Medicine. She has since held roles at IMS Health (now IQVIA) delivering a broad range of HEOR projects and at GSK heading the global economic modelling function. She is currently Senior Director at Gilead Sciences, leading economic modelling and health technology evaluations at the Centre of Excellence in Global HEOR.
Laura Dormer, BSc
Becaris Publishing, Royston, HRT, United Kingdom
Laura Dormer is Co-Founder and Editorial Director of Becaris Publishing Ltd, publisher of the Journal of Comparative Effectiveness Research and The Evidence Base website. Laura has worked in STM publishing for 20 years, and has launched and developed numerous successful journals, including JCER, which, over the past 10 years, has led to her particular interest in the fields of health policy, HEOR and RWE. In recent years she has dedicated her time to projects relating to her key areas of interest, including industry practice, publication ethics, patient engagement and plain language content.
Nikolaos Takatzoglou, PhD Candidate, MSc
Johnson and Johnson MedTech, Athens, A1, Greece
"Nikolaos Takatzoglou holds pivotal roles as the Health Economics and Market Access Lead for Greece & Cyprus and EMEA HEMA Lead for Wound Closure and Healing at Johnson & Johnson. With a dedicated decade at J&J, he's spearheading two global Generative AI POCs for HEMA. In academia, Nikolaos secured an MSc in Business Administration from Athens University of Economics and Business and an MSc in HTA from the University of Sheffield. He's actively pursuing an MSc in Data Analytics from the University of Glasgow and a PhD on Machine Learning for HTA at Erasmus University Rotterdam."
Use of Real-World Data and Evidence (RWD/E) to Support Evidence Gaps in HTA Submissions on Medical Devices When Direct Comparative Evidence Is Not Available
In high-risk Medical Devices (class IIb and III), Randomized Controlled Trials (RCTs) are not always an option due to various reasons such as ethical standards, problems with randomization or blinding, etc. As comparative effectiveness is not required for regulatory approval, clinical comparative evidence in Medical Devices (MDs) is rare and therefore often not fulfills the HTA authority’s demands. On the one hand market access is granted, and the devices are used in routine care, on the other hand the informed choice of patients and professionals is still limited. Where does the comparative evidence come from, as demanded/needed in HTA submissions? How can real world data (RWD) be used to close this evidence gap? In this forum, Dr. Petra Schnell-Inderst will open the session by providing a brief overview of the upcoming topic. Dr. Frank Hulstaert will address key evidence gaps in HTA submissions on medical devices when direct comparative evidence is not available. Professor Piotr Henryk Skarżyński will explain the current standard in pre- and post-market clinical study performance on MDs. Dr. Michael Urban will assess the challenges from the industry perspective on HTA submissions when direct comparative evidence is not available.
Moderators
Petra Schnell-Inderst, MPH, PhD, Dipl. Biol
UMIT TIROL - University for Health Sciences and Technology, Hall i. T., 7, Austria
Dr. Dipl.-Biol. Schnell-Inderst, MPH, is a Senior Scientist at the Institute of Public Health, Medical Decision Making and Health Technology Assessment at UMIT TIROL - University for Health Sciences and Technology in Hall i. T., Austria. She heads the Program on Health Technology Assessment at the Institute.
Her main research interests are in health technology assessment methodology with a focus on medical devices (see www.medtechta.eu. www.core-de.eu), digital health technologies, and public health interventions (screening programs).
Speakers
Frank Hulstaert, MD, MSc
Federal Centre for Health Care Knowledge (KCE), Brussels, Belgium
Frank Hulstaert studied medicine in Ghent (MD, 1985), informatics in Brussels (MSc) and biostatistics at Hasselt University, Belgium. He started a residency in internal medicine but soon turned to industry, where he worked at Applied Artificial Intelligence, IBM, Becton and Dickinson, Sandoz/Novartis and Innogenetics. He was mainly involved in the development of in vitro diagnostics, pharmaceuticals and biopharmaceuticals, as medical expert and later as medical director. He joined the Belgian Health Care Knowledge Centre (KCE) in 2004. As a senior researcher he is mainly involved in health technology assessments and since 2016 also in KCE Trials, a program of publicly funded practice-oriented trials. During the covid pandemic he coordinated the non-commercial covid clinical trials in Belgium.
Piotr H. Skarzynski, MD, PhD
Medical University of Warsaw, WARSAW, MZ, Poland
Prof. Piotr H. Skarzynski, MD, PhD, MSc realises his work in the World Hearing Center of Institute of Physiology and Pathology of Hearing and Institute of Sensory Organs. Specialist in ENT, pediatric ENT, audiology and phoniatrics, and public health. Participated in the 3rd Stakeholders Consultation meeting during which the World Hearing Forum of WHO was announced. A member of the Roster of Experts on Digital Health of WHO, President-elect of International Advisory Board of AAO-HNS and more. Interested in different aspects of hearing loss treatment, including steroids and pharmacoeconomics.
Michael Urban, Dr. rer. nat. MBA, MSc
Vibrant MED-EL Hearing Technology GmbH, Innsbruck, Austria
Dr. Michael Urban, MBA (MCI), MSc
Michael is a Group Leader Technology Assessment at MED-EL Medical Electronics. He is based in Innsbruck, Austria. Michael received his degree in Chemistry at the LMU Munich. After working in the bioanalytic field for more than 10 years, further education led him to an MBA in General Management at the Management Center Innsbruck and an MSc in Pharmacoeconomics and Health Economics at the UPF, Barcelona. Since 2012 he has been responsible for the reimbursement of Middle Ear & Bone Conduction Implants within the family of MED-EL’s implantable solutions. Based on his in-depth experience in clinical epidemiology, evidence-based medicine, health economics and public health, Michael focusses on planning, developing, reporting results of systematic literature reviews, and value dossiers, and communicating these outcomes to all kinds of stakeholders. He has successfully managed several submissions to reimbursement and HTA authorities worldwide.
Educational Symposia
Two concurrent sessions
15:30 - 16:15
Poster Tours
Pricing Reimbursement Poster Tour
Posters featured in this tour:
PT19: Analysis of the Access to Medicines for Rare Diseases in Countries from the Balkan Peninsula: A Comparative Analysis
PT20: Applications for Tolvaptan (JINARC®) Under a Managed Access Protocol in Ireland
PT21: Evaluation and Reimbursement of Digital Therapeutics in Germany, France, Belgium and England
PT22: Exploring Reimbursement Disparities: A Comparative Study of Pharmaceutical Access in Hong Kong and Mainland China
PT23: Is It Possible to Estimate the Welfare Economic Loss to Society of Not Having Value-Based Differential Pricing for Multi-Indication Pharmaceuticals: An Empirical Analysis in Denmark, Norway, and Sweden
PT24: Reimbursement Decisions in Ireland; Utilising Text Mining to Assess Factors Contributing to Decision Making
Sponsor
Moderator
Olivia Wu, PhD
University of Glasgow, Glasgow, United Kingdom
Olivia Wu, PhD is Director of the Health Economics and Health Technology Assessment (HEHTA) Research Unit and William R Lindsay Chair of Health Economics, at the University of Glasgow. She is also Director of the Complex Reviews Support Unit, a national methods support unit for evidence synthesis funded by the UK National Institute for Health Research (NIHR). Olivia has expertise in a broad range of health technology assessment (HTA) methodologies and is interested in adapting and applying HTA methodologies in context. She has a particular interest in evidence synthesis and economic evaluations. Her research spans across a wide range of clinical areas and different types of health technologies (eg, pharmacological treatments, medical devices and diagnostic tests). Her work has informed clinical guidelines and health policy decisions, both at national and international levels. In addition to her research, Olivia had been a long-standing member of the NICE Technology Appraisal Committee. Currently, she is advisor to HTA agencies in Scotland, the Philippines, Taiwan and Thailand.
Methodology Research in HEOR Poster Tour
Posters featured in this tour:
PT13: An Evaluation of the Impact of Evidence Grouping on Certainty Rating When Performing a Grade Assessment Without a Meta-Analysis
PT14: Bayesian Meta-Analysis of Data From Trials of Mixed Patient Populations to Inform Health Economic Models for Patients Harboring a Predictive Biomarker
PT15: Comparing the Impact of Random Forest Vs Bayesian G-Computation on Matching-Adjusted Indirect Comparisons of Treatments Between Trials: A Simulation Study
PT16: Impact of Data Maturity on the Estimation of the within-Trial Hazard Function: An Example from Metastatic Castration Resistant Prostate Cancer
PT17: Modelling Treatment Duration in Psoriasis: Examining Extrapolation Approaches and Implications
PT18: Novel Approaches to Unify Multi-State Transition Modelling Methods and Develop a Synthetic English Population to Support Health Economic Evaluation Studies
Sponsor
Moderator
Koen Degeling, PhD
Lumen Value & Access, Hengelo, OV, Netherlands
Dr Koen Degeling is Scientific Director and the Decision-Analytic Modeling Lead at Lumen Value & Access, a Healthcare Consultancy Group company. He was trained as an Industrial Engineer specializing in Healthcare Technology and Management, and holds a PhD in Health Economic Modelling from the University of Twente in the Netherlands. Prior to joining Lumen Value & Access, Koen worked on real-world data-driven health economic and health services research projects at the Cancer Health Services Research department of the University of Melbourne in Australia, where he continues to be involved as an Honorary Fellow. He is an active ISPOR member and currently serves on the Editorial Advisory Board for Values & Outcomes Spotlight and ISPOR New Professionals Steering Committee, is involved in several short courses and workshops, and has served as global chair of the ISPOR Student Network.
15:30 - 18:30
Poster Session 2
Live
15:45 - 16:15
Exhibit Hall Theater
Will Local Market Access Needs Ever Be Met by Global Evidence Packages? LiveSLR to the Rescue
Digital Conference Pass
With EU HTA Regulation (EU) 2021/2282, a new vehicle for standardizing HTAs, pharmaceutical sponsors will soon confront multiple sets of evidentiary requirements - for clinical review by the Joint Clinical Assessment (JCA) and for pricing and access by each of the different national bodies. To date, sponsor Global Value Dossiers (GVD) have provided a core compendium of evidence that is then tailored for national (local)-level HTA review. With JCAs coming soon, can sponsors still rely on GVDs to efficiently capture the abundance of clinical and economic data to meet the diverse and specific requirements for the JCA and for each local market?
Regularly updating evidence closer to local submissions along meeting specific population, intervention/comparator, outcomes, and study design (PICOS) requests for each local market remain significant challenges for companies preparing for multiple country submissions that may seriously compromise market access success. Furthermore, these challenges are magnified by a general push by decision-makers for faster patient access to promising health technologies that put pressure to companies to prepare submissions at higher evidentiary standards in a shorter preparation period. This is where LiveSLR® comes to the rescue; we will introduce how an online tool, LiveSLR® which uses automation and by adhering to HTA standards, can store and present the vast quantity of literature as part of a global evidence package, but can easily and transparently filter local relevant evidence based on PICOS and geography. The output from PICOS selection is an automated report with customized study selection criteria, tailored Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) flow of literature, with data visualization and graphics. By speeding up time-consuming steps in local submissions such as conducting or updating local systematic literature reviews, manufacturers can divert investment in time and resources towards stronger, value-based local market access submissions.
Sponsored by Cytel
Sponsor
Cytel
Moderators
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, United Kingdom
Grammati has 15+ years’ experience in epidemiology studies, evidence synthesis and decision making from her prior roles in academia, health technology assessment (NICE, UK) and private health consultancies. Grammati has strong methodological experience in generating impactful evidence from different sources (clinical trials, real-world evidence, economic studies), clearly communicating findings in an impactful way and using complex analytical methods for health care-decision making. Grammati currently holds the position of Chair of the ISPE comparative effectiveness research special interest group and is a topic co-editor for the Journal of Frontiers in Pharmacology. Grammati has numerous presentations (both as a moderator and panelist) in previous ISPOR conferences. For the particular topic, she has published a white paper https://pubmed.ncbi.nlm.nih.gov/35784688/ which was widely communicated and presented initial results of an ISPE funded project on unraveling health disparities in COVID-19 treatments at an issue panel at ISPOR US 2023.
Speakers
Julia Gaebler, PhD
Cytel, Cambridge, MA, USA
Julia Gaebler, PhD, brings more than two decades of experience in Global Market Access, Pricing, HEOR, RWE, Health Policy, Business Development and Corporate and Commercial Strategy in biopharma and consulting and in neurology, cardiology, cardiometabolic, oncology, hematology, and infectious and rare diseases. Prior to joining Cytel, Julia was CBO at Lucy Therapeutics, VP of Commercial Strategy at Milestone Pharmaceuticals, and Partner at Health Advances, a life-sciences strategy consulting firm. She has also held positions at Hoffmann-La Roche, Amylin, and Biogen, where she managed the global market access launches for FAMPRYA and TECFIDERA. Julia earned a PhD.in health policy and decision sciences from Harvard University, an MA in international economics from Johns Hopkins University School of Advanced International Studies, and a BA from the University of Pennsylvania.
Maria Rizzo, MSc
Cytel, London, London, United Kingdom
Maria Rizzo is a Vice President at Cytel with over 15+ years of relevant experience in evidence synthesis. Maria started her career conducting SLRs to inform clinical guidelines in mental health for the National Institute for Health and Care Excellence. She then spent over 12 years working in consultancy to support pharmaceutical companies navigate the reimbursement pathway. Maria specializes in considerations on PICOS selection to match the treatment pathway for reimbursement application.
16:15 - 16:45
Fast Facts
The EU Legislative Mountain is Scary: What Do HEOR Professionals Need to Climb It?
Since the COVID-19 pandemic, the EU has taken a larger role in securing access to lifesaving medicines for citizens in EU-27. The division between EU and national competencies is clearly described in the EU treaties and EU Member States are responsible for national pricing and reimbursement. However, every aspect of bringing a new medicine to market is impacted by EU legislation - from identifying study cohorts to pricing negotiations. New developments in the legislation on digitalization of healthcare, as well as the new Pharma Package, will impact the way in which HEOR is developed and used. Access and affordability will now take a front row seat in the discussions between regulators and industry at the point of marketing authorization, what does this mean for HEOR?
This session will provide a high-level map of the EU regulatory mountain range in the healthcare and life sciences sector. It will set out some of the challenges that HEOR professionals, clinical researchers and market access colleagues have to face, and how those challenges might be approached. Join us for a 30 min bird’s eye view and stay for discussion with two leading experts in EU pharmaceuticals regulation and policy.
Discussion Leader
Katja Murray, Master
FTI Consulting, Brussels, Belgium
Worked in various European roles in communication and policy communication and access policies in pharmaceutical industry
Petra Wilson, PhD
Health Connect Partners, FTI Consulting, Brussels, Belgium
Petra Wilson is co-founder and managing director of Health Connect Partners, a boutique consultancy which focusses primarily on helping clients understand the European health policy environment. In addition, Petra is acts as EU Policy Advisor to HIMSS and FTI Consulting and is a member of the team at the Institute for Innovation through Health Data, focusing on data governance. Alongside these roles Petra serves on the WHO’s Digital Health Technical Advisory Group; is a Board Member of the European Association for the Study of Diabetes (EASD), and serves on the Ethics Boards of several EU funded projects. Petra holds a Doctorate in Public Health Law from Oxford University, she has British and Belgian nationality and and works in English, German and French.
Petra’s professional experience includes eight years in the European Commission, where she focussed particularly on the use of information society technologies in healthcare; seven years as Senior Director of Connected Health at Cisco, where Petra’s team supported clients in making best use of new communications technologies to drive safer and more efficient access to healthcare. Petra also has deep experience of the health services sector, having worked on both the patient and provider side as CEO of the International Diabetes Federation; as well as experience in academia having been member of the teaching staff of both the law school and medical school of Nottingham University
16:15 - 17:15
Member Group Meetings
Global Access to Medical Innovation Special Interest Group Open Meeting
Presenting ISPOR’s newest Special Interest Group – the Global Access to Medical Innovation (GAMI SIG) excited to kick off its upcoming projects and interact with ISPOR members on shared interest for future activities! Join leaders of this SIG in planning webinars, journal clubs, and conference presentations for the upcoming year and explore ways to get more involved. Your participation and insights in guiding the SIG’s key projects are highly valuable! To view our SIG webpage, visit
here . The purpose of this SIG is:
to educate and inform ISPOR members and stakeholders on the basics of global access, especially as it relates to the role of HEOR across health systems, as well as new methodological and policy developments, and
to leverage the high motivation and skills of ISPOR’s global community of researchers, patient representatives, and other life science professionals to improve global access to medical innovation while stimulating its production.
We look forward to seeing you at the GAMI SIG Open Meeting!
ISPOR Brazil Chapter Meeting
This meeting is open to anyone interested in connecting with the ISPOR Brazil chapter. The chapter will provide a brief report on their local experience with educational meetings, such as the local charter meeting, and discuss upcoming projects and activities.
16:45 - 17:30
ISPOR Education Center in Action
Stop by for a demo of the newly launched ISPOR Education Center that provides instant access to HEOR education with on-demand programs that you can work through at your own time and pace. Drive your professional development with ISPOR's innovative course curricula and personalized, powerful learning platform—let us show you how!
17:00 - 18:00
Case Studies
Pricing and Procurement Case Studies
These case studies are focused on pharmaceutical pricing and access policies. They cover topics such as international reference pricing, digital platforms for contract management, alternative pricing arrangements for rare disease treatments, and value-based procurement in healthcare. The studies highlight the importance of policy development, methodological considerations, and innovative approaches to enhance patient access to pharmaceutical products and treatments in different healthcare systems.
CS7: Alternative Pricing Arrangements as an Opportunity for Broader Access to Rare Disease Treatments: Case Study for an Outcomes-Based Agreement for Nusinersen in Norway
5:30PM - 5:45PM
Gómez Castañeda M1 , Torup Østby J 2 1 Biogen Norway AS, Oslo, Norway, 2 Biogen Norway AS, Oslo, 03, Norway
Problem Statement: Treatments for rare diseases can pose challenges for payers relying on cost-effectiveness evaluations. Small patient populations, short follow-up time for treatment effect, and uncertainty associated with new treatment mechanisms, are some of the challenges that complicates decision making. Alternative pricing strategies such as outcomes-base agreements (OBA), have the potential to reduce some of this uncertainty by making the financial decision partially reversible and by facilitating further generation of real-world evidence.
Description: A framework for advancing OBA proposals for the Norwegian Hospital Procurement Agency was established in 2020. Based on this, an OBA to expand access for adult SMA patients to nusinersen treatment was developed and implemented in May 2023, being the first OBA of such complexity in Norway. It is a five-year agreement where motor- and respiratory functions and disease progression are measured systematically in all treated patients during this period. Outcomes are registered by treating physicians in a national registry. A pre-defined scoring system for different treatment outcomes determines on a group level whether price will change at the end of the agreement period in the following way: 1) Stable disease = No change in price, 2) Significant disease improvement = Increased price, 3) Significant deterioration = Decreased price. All outcomes are measured as group-level averages.
Lessons Learned: OBA has proved to be a viable approach to provide access to treatments associated with high decision uncertainty. Early dialogue with payers, clinicians and registry holders are crucial to secure effective execution of such agreements. A predefined submission framework for OBA and a centralized decision-making body and registry, are also central for agreements of this complexity to be successful.
Stakeholder perspective: Industry perspective
CS8: The Value-Based Procurement Application in Dialysis: A Kingdom of Saudi Arabia (KSA) Case Study
5:45PM - 6:00PM
Metwally I 1 , Lima F 2 , Busink E 3 1 Fresenius Medical Care Kingdom of Saudi Arabia, Riyadh, 01, Saudi Arabia, 2 Fresenius Medical Care Deutschland GmbH, Milano, Italy, 3 Fresenius Medical Care Deutschland GmbH, Bad Homburg, Germany
Problem Statement: Misalignment in procurement practices is a significant challenge in healthcare. It leads to 1) a limited consideration for the most economically advantageous tender (MEAT), 2) a slowing adoption of innovation, 3) short-term contracts, 4) intransparency in costs and 5) a reduction of patient value. Value-Based Procurement (VBP) is a multidisciplinary approach to address unmet needs, to achieve better outcomes and cost-effective care resulting in MEAT. Total Cost of Ownership (TCO), as part of the VBP, allows to understand the cost impact and optimal cost planning for healthcare institutions. Here we report on one example of VBP in dialysis in the KSA.
Description: A TCO analysis was performed:
assessing the current set-up and how different changes could address unmet needs
evaluating the switch from eight suppliers for hemodialysis and acute dialysis to one partner
Lessons Learned: The analysis showed the cost drivers and how the switch to one partner could be a solution for the customer’s unmet needs (low water quality, no hemodiafiltration, high cost of consumables and maintenance). The TCO demonstrated that even with the investment for upgrading the installation base, a saving of 32% over 2 years was achieved (mainly due to less failures and lower maintenance costs of the change in supplier). Therefore, also the administrative/operational resource burden for the hospital is significantly reduced.
Stakeholder perspective: The analysis demonstrated that cost savings on the total treatment cost can be made while providing high-quality dialysis. TCO produces a win-win situation for both the supplier and hospital. It facilitates value-based purchasing, thereby enabling cost-effective care with the potential of a positive financial impact and it allows to demonstrate the supplier as a reliable partner to tackle the challenges and to implement the solutions.
CS6: Automating the Management of Innovative Contracts Through a Digital Platform in Ireland to Support Patient Access
5:15PM - 5:30PM
Burnham-stevens C1 , McCarthy D 2 , Sullivan J3 , Stephens C2 , Blezat A 4 1 Ernst & Young LLP, London, London, UK, 2 Roche, Dublin, Dublin, Ireland, 3 Roche, Dublin, Ireland, 4 Ernst & Young LLP, London, UK
Problem Statement: Innovative contracts have long been hailed as a solution to improve patient access to medicines. However, the challenges associated with their management have prevented their widespread adoption and ability to scale.
Description: In order to enable greater access to an innovative medicine for a rare disease, a pharmaceutical company and a hospital in Ireland agreed an innovative complex pricing agreement which would enable access to the Irish patient population. However, as the patient population increased, the administrative burden of managing the contract became increasingly time-consuming and complex from a data extraction, calculation and adjudication perspective. To support the management of the contract, the pharmaceutical company and the healthcare provider agreed to explore the use of digital tools to automate the process and perform the previously manual data transformation, data validation and calculations. Through the use of a digital, cloud-based platform to automate these processes, the benefits were as follows: the hours saved across the two organisations amounted to >15 days per month, the tool reduced the delays in rebate calculations, increased data validations in order to identify and flag any potential errors and enabled the scaling of the contract to a larger patient population. The use of the platform also allowed for the transformation of data from >80 documents to a single source, whilst remaining compliant with GDPR and data privacy. The insights generated from the platform also enabled both the provider and the pharmaceutical company to understand better the product usage, patient population and identify additional trends within the patient population.
Lessons Learned: Innovative pricing arrangements can be used to support increased patient access, and with the use of digital tools challenges associated with these types of agreements can be mitigated, enabling these models to scale and ultimately benefit more patients.
Stakeholder perspective: Industry
CS5: International Reference Pricing Policy of Pharmaceutical Products in Saudi Arabia – Policy Development and Methodological Considerations
5:00PM - 5:15PM
Al Hussain F Almaghrabi M
Problem Statement: International Reference Pricing (IRP) is a price regulation tool widely used by policymakers to set drug price in the country considering its price in other country. IRP implementation might be problematic thereby policymakers need to carefully select the basket countries to achieve the intended outcomes. However, IRP implementation process or country experience is scarce in the literature.
Description:
Lessons Learned:
Stakeholder perspective:
Breakouts: IP, WS, & OBS
Filling HTA Regulation Implementation Knowledge Gaps – Q and A on Identified Gaps in Currently Available Information on Joint Clinical Assessment and Joint Scientific Consultation From EUnetHTA21 and in the Regulation Itself
Digital Conference Pass
Level: Intermediate
PURPOSE: The objective of this session is to address significant knowledge gaps that exist regarding the practical implementation of the EU Regulation on Health Technology Assessment (HTAR) as it pertains to health technology developers (HTD)s. DESCRIPTION: The HTA regulation will apply to pharmaceutical and medical device companies in a stepwise fashion beginning 12 January 2025. It includes mandatory EU-level assessment of clinical relative effectiveness and safety, called Joint Clinical Assessments (JCA)s, and optional advice procedures, called Joint Scientific Consultations (JSC)s. With a little over a year to go, many practicalities of the implementation are still unknown, hampering preparation by the HTDs. In this session, Dr Sigrid Klaar will interview Niklas Hedberg, former chair of EUnetHTA 21 and current co-chair of The HTA Coordination Group (HTACG), and Anne Willemsen, co-chair of the JCA subgroup under the HTACG, in a structured way, based on uncertainties identified in systematic review of the HTAR and EUnetHTA 21 deliverables. Audience participation is invited and encouraged. Issues such as the timelines of the JCA, the number of PICOs (the analysis scope) and handling of dossier requirements, HTD interaction with assessors during JCA, the selection of medical devices to undergo JCA, and the availability and prioritization of JSCs will be discussed and clarified. This session will benefit a broad range of industry professionals who will be affected by the new Regulation.
Discussion Leaders
Sigrid Klaar, MD, PhD
NDA Group, Uppsala, C, Sweden
MD, PhD, Specialist in Oncology. Medical Advisor and NDA Group Advisory Board member.
Ex-regulator and ex-HTA.
Previously senior clinical assessor at the Swedish Medical Products Agency with extensive experience from marketing authorisation applications in the EU centralised procedure and scientific advice. Vice-chair of the EMA Oncology Working Party, drafting guidelines to industry and supporting the CHMP on oncology issues.
Previously also Medical Advisor at the Swedish Dental and Pharmaceutical Benefits Agency (TLV), with experience from reimbursement applications and EUnetHTA joint clinical assessments, and HTA regulation deliberations.
Discussants
Niklas Hedberg, MSc
The Dental and Pharmaceutical Benefits Agency (TLV), Stockholm, AB, Sweden
Niklas Hedberg is the Chief Pharmacist
at the Swedish governmental authority,
the Dental and Pharmaceuticals Benefits
Agency (TLV).
Niklas is the co-chair for medicinal products in the European HTA Coordinaton Group.
Niklas was the chair of the EUnetHTA JA3 and EUnetHTA21 Executive Board from 2018 to 2023.
Niklas has been involved in the national
pricing and reimbursement decision
making for pharmaceutical products
since 2001, and for medical devices
since 2009. He has a broad experience in
different aspects of value-based
evaluation.
Anne Willemsen, MSc
Zorginstituut Nederland (ZIN), Diemen, Netherlands
Anne Willemsen works for the Dutch National Healthcare Institute (Zorginstituut Nederland) and has been coordinating Joint Clinical Assessments (JCA) under EUnetHTA Joint Action 3 and EUnetHTA 21. In April 2023, she was elected as the co-chair of the HTA Coordination Group JCA subgroup under the HTA Regulation.
Podium Sessions
Expanding Data Sources: Creative Use of Data in Health Economics Analyses
Digital Conference Pass
Moderator
Seamus Kent, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Seamus is Assistant Professor in HTA at Erasmus University Rotterdam. Prior to joining Erasmus Seamus was a Senior Adviser in Data & Analytics at NICE where he led the development of NICE's Real-world evidence framework and a Senior Adviser in HTA at Flatiron Health a RWD company in oncology.
P18: Adjusted Comparison of Teclistamab Versus Real-World Physician’s Choice (RWPC) of Therapy in Patients with Triple-Class Exposed (TCE) Relapsed/Refractory Multiple Myeloma (RRMM)
5:15PM - 5:30PM
Mateos MV1 , Chari A2 , Usmani SZ3 , Goldschmidt H4 , Weisel K5 , Qi K6 , Londhe A6 , Nair S 7 , Lin X8 , Pei L9 , Ammann E10 , Chastain K9 , Parekh T11 , Marshall A10 , Slavcev M10 , Moreau P12 1 University Hospital of Salamanca/IBSAL/CIC/CIBERONC, Zamora, ZA, Spain, 2 Mount Sinai School of Medicine, New York City, NY, USA, 3 Memorial Sloan Kettering Cancer Center, New York, NY, USA, 4 Internal Medicine V, GMMG-Studygroup at University Hospital Heidelberg, Heidelberg, Germany, 5 University Medical Center Hamburg-Eppendorf, Hamburg, Federal Republic of Germany, Germany, 6 Janssen Research & Development, Titusville, NJ, USA, 7 Janssen Pharmaceutica NV, Beerse, VAN, Belgium, 8 Janssen Global Services, Horsham, PA, USA, 9 Janssen Research & Development, Raritan, NJ, USA, 10 Janssen Global Services, Raritan, NJ, USA, 11 Janssen Research & Development, Bridgewater, NJ, USA, 12 Hematology Clinic, University Hospital Hôtel-Dieu, Nantes, France
OBJECTIVES: Patients with RRMM who receive ≥3 prior lines of therapy (LOT) have limited treatment options. Teclistamab is the only approved BCMA×CD3 bispecific antibody with a personalized, weight-based dosing schedule for the treatment of TCE RRMM. We assessed the comparative effectiveness of teclistamab vs RWPC of therapy.
METHODS: Individual patient-level data for patients who received teclistamab (1.5 mg/kg subcutaneous QW, Q2W, or Q4W; clinical cut-off: Jan 4, 2023) in the phase 1/2 MajesTEC-1 trial (NCT03145181/NCT04557098) were compared with an external control arm. The control arm comprised patients treated with RWPC as subsequent therapy after discontinuing study treatment from 4 daratumumab trials (CASTOR, POLLUX, EQUULEUS, APOLLO) who met key eligibility criteria for MajesTEC-1 (N=427 unique patients; 806 observations). Inverse probability of treatment weighting (IPTW) was used to adjust for imbalances in baseline covariates. Outcomes of interest included ORR, ≥VGPR rate, TTNT, PFS, and OS. For binary endpoints, a weighted logistic regression method was used to derive an OR and 95% CI. A weighted Cox proportional hazards model was used to compute HRs and 95% CIs for time-to-event endpoints. A sensitivity analysis, including additional covariates, was also performed.
RESULTS: After IPTW adjustment, baseline characteristics were comparable between cohorts. Patients had significantly improved outcomes with teclistamab vs RWPC of therapy: ORR (OR, 4.80; 95% CI, 3.03–7.70; P <0.0001), ≥VGPR rate (OR, 12.37; 95% CI, 7.08–22.65; P <0.0001), TTNT (HR, 0.32; 95% CI, 0.24–0.41; P <0.0001), PFS (HR, 0.58; 95% CI, 0.45–0.75; P< 0.0001), and OS (HR, 0.52; 95% CI, 0.39–0.68; P <0.0001). Results of the sensitivity analysis were statistically significant, in favor of teclistamab, and consistent with the primary analysis described above.
CONCLUSIONS: Using updated MajesTEC-1 data, these analyses demonstrated significantly improved effectiveness with teclistamab vs RWPC of therapy, highlighting its clinical benefit in patients with TCE RRMM who have limited treatment options.
P20: Matching-Adjusted Indirect Treatment Comparison (MAIC) of Teclistamab Vs Belantamab Mafodotin for the Treatment of Patients with Triple-Class Exposed (TCE) Relapsed/Refractory Multiple Myeloma (RRMM)
5:45PM - 6:00PM
Moreau P1 , Usmani SZ2 , van de Donk NWCJ3 , Garfall AL4 , Delforge M5 , Oriol A6 , Nooka A7 , Rosiñol L8 , Bahlis N9 , Rodríguez Otero P10 , Martin TG11 , Diels J12 , Van Sanden S 12 , Pei L13 , Ammann E14 , Chastain K13 , Marshall A14 , Slavcev M14 , Londhe A15 , Krishnan A16 1 Hematology Clinic, University Hospital Hôtel-Dieu, Nantes, France, 2 Memorial Sloan Kettering Cancer Center, New York, NY, USA, 3 Amsterdam University Medical Center, Vrije Universiteit Amsterdam, Amsterdam, Netherlands, 4 Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA, 5 Department of Haematology, University of Leuven, Leuven, Belgium, 6 Institut Català d’Oncologia and Institut Josep Carreras, Hospital Germans Trias i Pujol, Badalona, Barcelona, Spain, 7 Winship Cancer Institute, Emory University, Atlanta, GA, USA, 8 Hospital Clinic, IDIBAPS, University of Barcelona, Barcelona, Spain, 9 Arnie Charbonneau Cancer Institute, University of Calgary, Calgary, AB, Canada, 10 Clínica Universidad de Navarra, CIMA, CIBERONC, IDISNA, Pamplona, Navarra, Spain, 11 University of California San Francisco, San Francisco, CA, USA, 12 Janssen Pharmaceutica NV, Beerse, Belgium, 13 Janssen Research & Development, Raritan, NJ, USA, 14 Janssen Global Services, Raritan, NJ, USA, 15 Janssen Research & Development, Titusville, NJ, USA, 16 City of Hope Comprehensive Cancer Center, Irvine, CA, USA
OBJECTIVES: Teclistamab is the only approved BCMA×CD3 BsAb with a personalized, weight-based dosing schedule for the treatment of TCE RRMM. We assessed the comparative efficacy of teclistamab vs belantamab mafodotin for patients with TCE RRMM.
METHODS: An unanchored MAIC was performed using individual patient-level data (IPD) for patients who received teclistamab (1.5 mg/kg subcutaneous QW, Q2W, or Q4W; clinical cutoff: Jan 4, 2023; N=165) in MajesTEC-1 (NCT03145181/NCT04557098) and published summary‑level data from the 2.5-mg/kg cohort in DREAMM-2 (NCT03525678; N=97). IPD from MajesTEC-1 patients meeting DREAMM-2 eligibility criteria were included and weighted to match aggregated DREAMM-2 baseline characteristics (BCs). The analysis adjusted for BCs of prognostic significance (refractory status, cytogenetic profile, ISS stage, presence of extramedullary disease, and number of prior lines of therapy). Comparative efficacy was estimated for ORR, ≥VGPR rate, ≥CR rate, DOR, PFS, and OS. For binary endpoints, relative effects were quantified using ORs and risk ratios (RRs) with 95% CIs derived from a weighted logistic regression analysis. For time-to-event endpoints, HRs including 95% CIs were estimated using a weighted Cox proportional hazards model.
RESULTS: BCs for reweighted MajesTEC-1 patients were balanced with the DREAMM-2 population. Teclistamab demonstrated improved ORR (OR, 1.85 [95% CI, 0.88–3.89]; RR, 1.46; P =0.1049), ≥VGPR rate (OR, 3.35 [1.52–7.39]; RR, 2.33; P =0.0028), ≥CR rate (OR, 4.45 [1.76–11.20]; RR, 3.37; P =0.0016), DOR (HR, 0.27 0.13–0.58]; P =0.0008), PFS (HR, 0.63 [0.38–1.05]; P =0.0751), and OS (HR, 0.88 [0.55–1.41]; P =0.5906) vs belantamab mafodotin. Cross-trial differences in BCs led to a relatively low effective sample size (n=40) for MajesTEC-1 post-adjustment, resulting in wide CIs for some outcomes.
CONCLUSIONS: Using updated MajesTEC-1 data, these analyses demonstrated improved efficacy of teclistamab vs belantamab mafodotin for most outcomes, highlighting the clinical benefit of teclistamab in patients with TCE RRMM, a population with high unmet medical need.
P19: Demographics of Patients and Societal Burden of Obstructive Hypertrophic Cardiomyopathy: Results From a Real-World Survey in Italy and Spain
5:30PM - 5:45PM
Lowe M1 , Jackson J2 , Tebbs K2 , Zhong Y3 , Contente M4 , Barlow S2 , Krause T 5 1 Adelphi Real World, Bollington, Cheshire, UK, 2 Adelphi Real World, Bollington, UK, 3 Bristol Myers Squibb, Princeton, NJ, USA, 4 Bristol Myers Squibb, Uxbridge, London, UK, 5 Bristol Myers Squibb, LONDON, LON, UK
OBJECTIVES:
To describe the clinical characteristics, burden of illness, productivity, and support needs of patients with obstructive hypertrophic cardiomyopathy (HCM) in Italy (IT) and Spain (ES).
METHODS:
Between July 2022 and January 2023, cardiologists provided information on up to 10 consecutive adult patients with symptomatic obstructive HCM as part of the Adelphi Disease Specific Programme, a cross-sectional survey with partial retrospective data collection.
RESULTS:
118 cardiologists (60, IT; 58, ES) provided data for 725 patients with obstructive HCM (365, IT; 360, ES). For IT and ES respectively, mean [standard deviation] age was 55.7 [13.0] and 58.2 [15.9] years, 65.8% and 60.3% were male and mean time since diagnosis was 2.4 and 3.6 years. 17.3% and 16.7% had undergone septal reduction therapies and 91.0% and 86.7% were currently prescribed pharmacological treatments for HCM, in IT and SP respectively. Symptomatic burden was similar in both countries, with dyspnoea (when active), palpitations, and fatigue most commonly reported. For IT and ES respectively,
11.5% and 9.2% of NYHA II, and 30.6% and 30.4% of NYHA III patients were not working due to HCM. In IT and ES respectively, 46.9% and 38.7% of NYHA III and 85.7% and 100.0% of NYHA IV patients required carer support. Mean support hours per week were 33.9 (IT) and 51.5 (ES); most carers were unpaid family members (89.9% (IT) and 96.1% (ES)). CONCLUSIONS:
Obstructive HCM is associated with substantial clinical and societal burden. As NYHA increases, patient productivity decreases and need for carer support
increases . Despite current treatment, high symptom burden remains. This suggests the need for alternative treatment options.
P17: A Retrospective Analysis of Medicare Current Beneficiary Survey Data to Evaluate the Alzheimer's Disease and Related Dementia Risk-Reducing Benefits Offered By HMGCR Inhibitors or Statins
5:00PM - 5:15PM
Chanpura M 1 , Akincigil A2 , Khan Z1 1 Rutgers University, Piscataway, NJ, USA, 2 Rutgers University, New Brunswick, NJ, USA
OBJECTIVES: Alzheimer’s disease and related dementias comprise a class of fatal neurodegenerative conditions with few costly treatment options of dubious efficacy. In contrast, HMGCR inhibitors, or statins, comprise a well-established, low-cost class of effective treatments for dyslipidemia. As ADRD is thought to be an outcome of excessive cerebral Aꞵ aggregation triggered by dyslipidemia, statins are also believed to offer prophylaxis against ADRD. The objective of this analysis was to assess whether statins significantly reduce ADRD risk among older adults and, if so, which statin drugs offer the greatest risk reduction.
METHODS: A retrospective matched case-control analysis was conducted using 2019-2020 Medicare Current Beneficiary Survey data. Eligible cases of ADRD were identified via the MCBS Health Status Functioning Questionnaire and the Health Status section of the MCBS Facility Instrument. Each eligible case was matched with two controls based on sex, race, nationality, age stratum, and income level; respondents <45 and those missing corresponding prescription data were excluded. Statin use among included cases and controls was determined via the MCBS Prescription Medication Utilization Questionnaire. Associations between statin use and ADRD diagnosis were assessed via Fisher’s exact test using SAS® v9.4.
RESULTS: Statin use was found to significantly reduce risk of ADRD diagnosis (OR 0.68, 95% CI: 0.59-0.78; p<0.0001). Among individual statins, rosuvastatin was found to be significantly more effective at reducing ADRD risk than atorvastatin (OR 0.66, 95% CI: 0.47-0.94; p=0.0193). Hydrophilic statins were also found to be significantly more effective at reducing ADRD risk than lipophilic statins (OR 0.77, 95% CI: 0.60-0.98; p=0.0394).
CONCLUSIONS: Statin use was found to significantly reduce ADRD risk among the analyzed cohort of older adults. However, future longitudinal research remains necessary to confirm that statins are indeed an effective prophylaxis against ADRD.
Using Artificial Intelligence to Enhance Efficiency in Systematic Reviews and Meta-Analyses
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This session aims to delve into the transformative role of artificial intelligence (AI) in revolutionizing literature reviews and meta-analyses. The presentations will explore AI techniques for literature screening, data extraction, and analysis, offering insights into how these technologies can enhance the efficiency of evidence synthesis.
Moderator
Tamas Agh, MSc, PhD, MD
1) Center for Health Technology Assessment and Pharmacoeconomic Research, University of Pécs, Pécs, Hungary 2) Syreon Research Institute, Budapest, PE, Hungary
Tamás Ágh MD MSc PhD DrHabil graduated from the Semmelweis Medical University (MD, 2006), the Corvinus University of Budapest (MSc Physician-Economist, 2010), the Semmelweis University School of PhD Studies (PhD in Pharmacoeconomics, 2013) and the University of Pecs Doctoral School of Health Sciences (DrHabil in Health Sciences, 2020). He specialized in family medicine in 2010. Dr. Ágh is based in Hungary and works as a research associate professor and the head of the Medication Adherence Research Group at the Center for Health Technology Assessment and Pharmacoeconomic Research at the University of Pecs. He is also a principal researcher at the Syreon Research Institute and has been practicing as a medical doctor since 2006. With a professional background spanning over 15 years, his areas of specialization in health economics and outcome research include medication adherence, patient-reported outcomes measures, health technology assessment, and evidence synthesis. He is the chair of the ISPOR Medication Adherence and Persistence Special Interest Group and holds leadership position in the European Network to Advance Best practices & technoLogy on medication adherencE (ENABLE) Cost Action. Dr. Ágh has co-authored about 50 peer-reviewed articles.
P21: A Comparative Analysis of Large Language Models (LLM) Utilised in Systematic Literature Review
5:00PM - 5:15PM
Rathi H 1 , Malik A2 , Behera DC2 , Kamboj G3 1 Skyward Analytics Pvt. Ltd., Gurugram, Haryana, India, 2 EasySLR Pvt. Ltd., Gurugram, Haryana, India, 3 Skyward Analytics Pvt. Ltd., New Delhi, DL, India
OBJECTIVES: The objective of this study was to conduct a comparison of three Large Language Models (LLMs) — AI21 Ultra, OpenAI GPT-4, and Google Vertex Artificial Intelligence (AI) Model Bison in their application to primary screening during a systematic literature review (SLR).
METHODS: We fed three to five sample responses and identical screening rules to all LLMs for primary screening (title and abstract screening) of 100 studies. We compared the decision made by the human reviewer, assumed as reference response, to gauge the performance of the LLMs. Models were assessed on decision match rate (defined as cases where inclusion and exclusion decisions were identical between the human reviewer and LLM) and sensitivity score (defined as the number of correct inclusions by LLM to overall inclusions by the human reviewer).
RESULTS: Model Bison, GPT-4, and AI21 Ultra scored a decision match rate of 67.01%, 65.56%, and 64.0%, respectively. Model Bison had the highest sensitivity score of 0.90, followed by 0.74 and 0.71 for GPT-4 and AI21 Ultra, respectively. In scenario analysis, we noted that the performance metrics of all LLMs varied substantially based on the amends in screening rules, the number of sample responses fed, and the number of studies analysed. While the decision match rate dropped to 51% in a few scenarios, the sensitivity score increased to 0.97 in others.
CONCLUSIONS: The results highlight LLMs' potential to assist with the SLR process. All three LLMs were comparable in decision match rate metric, whereas in our simulation, Model Bison showed better sensitivity score than GPT-4 and AI21 Ultra. However, the results should be interpreted cautiously as the results may vary with different research questions. Future research should consider analysing the performance of LLMs on larger datasets, variation in number of sample responses fed, and calibration around framing of screening rules for better understanding by AI.
P24: Improving Efficiency of Living Systematic Literature Reviews (SLR) with Artificial Intelligence (AI): Assisted Extraction of Population, Intervention/Comparator, Outcome, and Study Design (P-I/C-O-S)
5:45PM - 6:00PM
Liu R 1 , Jafar R2 , Girard LA3 , Thorlund K4 , Rizzo M5 , Forsythe A6 1 Cytel Inc., Toronto, ON, Canada, 2 Cytel Inc., Vancouver, BC, Canada, 3 Cytel Inc., Montreal, QC, Canada, 4 McMaster University, Hamilton, ON, Canada, 5 Cytel, Kent, KEN, UK, 6 Cytel, Waltham, MA, USA
OBJECTIVES: SLRs are crucial but time-consuming parts of any evidence-based research. SLR protocols’ modifications add further complexity. We developed an AI-assisted tool, LiveSTARTTM , which extracts P-I/C-O-S information from citations during SLR review.
METHODS: LiveSTARTTM utilizes a fine-tuned generative biomedical large-language model to identify P-I/C-O-S-relevant texts from citations (title+abstract) helping reviewers identify the information necessary for acceptance/rejection decision based on inclusion/exclusion criteria. The model was trained using in-house annotated datasets consisting of 1400 citations, spanning 18 oncology and 6 non-oncology indications from clinical, economic, and health-related-quality-of-life (HRQoL) SLRs.
The model accuracy was evaluated by two human reviewers who scored a separate test dataset with 350 LiveSTART
TM predictions against manually identified P-I/C-O-S using 3 categories: completely correct, completely incorrect, or partially correct. We defined two sets of evaluation metrics: 1) The strict score considered partially correct predictions as incorrect. 2) The lenient score considered partially correct predictions as correct. Additionally, we evaluated the time savings LiveSTART
TM P-I/C-O-S model provides when an SLR protocol changes midway through projects.
RESULTS: The strict accuracy for P-I/C-O-S extraction were 93%, 89%, 45%, and 97%, respectively. The lenient accuracy were 99%, 96%, 98%, and 98%, respectively. The average strict accuracy was 81%; the average lenient accuracy was 98% . The lowest score was related to the strict accuracy for Outcome extraction (45%). The model could generally predict most of outcome measures in citations (with a lenient accuracy of 98%) which we found to be sufficient to assist reviewers in decision-making. When one of the P-I/C-O-S inclusion/exclusion criteria was changed, it took a human reviewer 4 hours to re-review manually, compared to 1 hour with the help of LiveSTART P-I/C-O-S annotation, yielding 75% in time saving.
CONCLUSIONS: The utilization of LiveSTARTTM to extract P-I/C-O-S shows high accuracy and significant time savings while dealing with protocol changes.
P22: Disrupting Health Economics: Automating Network Meta-Analyses With AI and Large Language Models
5:15PM - 5:30PM
Reason T 1 , Malcolm B2 , Klijn S3 , Langham J4 , Gimblett A1 , Benbow E1 1 Estima Scientific Ltd, London, UK, 2 Bristol Myers Squibb, Middlesex, LON, UK, 3 Bristol Myers Squibb, Lawrence Township, NJ, USA, 4 Estima Scientific Ltd, London, LON, UK
OBJECTIVES: The advancement of Large Language Models (LLMs), such as GPT-4, provides opportunities for automating data extraction and analysis in systematic reviews and meta-analyses. However, their practical application in Health Economics and Outcomes Research (HEOR) remains unverified. Our study aimed to evaluate GPT-4's accuracy in replicating a Network Meta-Analysis (NMA) result on overall survival of adult patients with advanced non-small cell lung cancer (NSCLC) post platinum-based treatment and pre-immunotherapy.
METHODS: Using GPT-4 through a Python API, we extracted survival data from the abstracts of eleven studies, transformed it to the log scale, and generated an R script for NMA using generic code from the R 'multinma' package. GPT-4 updated the code with new data and produced an executable R script that was run end-to-end in Docker, and parsed Docker output to create a mini NMA report. This was compared to the original human conducted NMA.
RESULTS: The LLM-generated model accurately replicated the overall survival outcome using abstracts and generic R code. It successfully extracted and converted survival data, created the NMA R script, ran it using Docker, and accurately produced the original NMA results. This was achieved with a single generic python script, demonstrating GPT-4's capability to perform end-to-end NMA using unstructured abstracts.
CONCLUSIONS: This study offers promising evidence for the potential of AI models like GPT-4 in automating data extraction and NMA. Further studies are necessary to confirm these findings in diverse contexts and investigate AI's potential in enhancing systematic reviews and NMA. Further exploration is also required on multimodal versions, and the ability of LLMs to validate the proportional hazards assumption.
P23: Evolving Use of Artificial Intelligence and Machine Learning in Systematic Literature Reviews (SLRs)
5:30PM - 5:45PM
Kamra S1 , Verma A2 , Pagidigummula R3 , Kohli IS2 , Goyal R4 , Kumar S2 , Rtveladze K 5 1 IQVIA, Gurugram, India, 2 IQVIA, Delhi, DL, India, 3 IQVIA, Mumbai, DL, India, 4 IQVIA, Thane, MH, India, 5 IQVIA, London , UK
OBJECTIVES: SLRs are fundamental to evidence-based decision making and often require screening thousands of articles. The increasing scientific data raises the need to incorporate artificial intelligence and machine learning (AI/ML) to analyse vast volume of data. Although there are many AI/ML tools that facilitate SLR process, there is lack of awareness in conducting AI/ML-enabled SLRs. The objective of current review was to analyse the trends and understand gaps of using AI/ML tools in published SLRs.
METHODS: Targeted searches were conducted on Embase® , MEDLINE® and Cochrane databases using OVID SP® on 07 Jun 2023. Search terms used were AI/ML, deep learning, SLRs and meta-analysis and specific AI/ML-enabled databases. No restrictions were applied on indication, geography and timeframe. Hand searches were also supplemented.
RESULTS: Thirty-seven SLRs used AI/ML-assisted databases such as Abstrackr® (11 SLRs), Rayyan® (10 SLRs), Covidence® (10 SLRs) and DistillerSR® (five SLRs) between 2015-23. Majority of identified AI/ML-enabled SLRs were conducted in United States and United Kingdom and used for title/abstract screening (35 SLRs), followed by full-text screening (nine SLRs) and data extraction (four SLRs).
The availability of AI/ML tools in conducting SLRs increased over the years, from one in 2015 to 11 in 2022 (5 by Mid-2023). Although availability has increased, AI/ML tool was used in only 37 SLRs between 2015-23 (4 by Mid-2023). Reasons of limited use of AI/ML tools in SLRs were: 1) Validity and reliability of most of AI/ML tools currently not established, 2) costs and training required and 3) lack of guidance on acceptability of automated SLRs by health technology assessment (HTA) bodies.
CONCLUSIONS: Availability of AI/ML tools and their use in published SLR has increased in past and might further increase with recent development in AI/ML tools like GPT-3. However, guidance from HTA bodies and increased experience of these tools is needed for growth of AI/ML in SLR.
Breakouts: IP, WS, & OBS
Using Performance Outcome Assessments in the Evaluation of Clinical Benefit in Multinational Treatment Trials: Unique Challenges and Considerations Along the Path From Selection to Implementation of a Fit-for-Purpose Measure
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Level: Intermediate
PURPOSE: This workshop builds on ISPOR task force recommendations for selection, modification, and/or development of performance outcome (PerfO) assessments by reviewing unique aspects of PerfO assessments relative to other clinical outcome assessment (COA) types and addressing how these differences impact the implementation of PerfO assessments in clinical trials.
DESCRIPTION: When evaluating clinical benefit of therapeutic interventions, it is critical that the outcomes assessed reflect aspects of health that are clinically meaningful to patients. Ideally, PerfO assessments are used when the optimal means of capturing clinical benefit is through the completion of defined tasks involving physical, cognitive, and/or sensory function that reflect activities that are meaningful in daily life. PerfO assessments are based on standardized tasks actively undertaken by a patient according to a set of instructions and administered by a trained individual or completed by the patient independently.
Although PerfO assessments share many of the same scientific and methodological considerations with the other COA types, they require special attention due to the nature of the relevant meaningful aspects of health, their relationship to concepts of interest, and the often indirect method of assessing them. PerfO assessments are frequently used in pediatric populations and patient groups with cognitive challenges, requiring additional considerations. Ms. Eremenco will chair the session and provide a brief overview of areas where PerfO assessments differ from other types of COAs with regard to their selection, modification, and/or development; Ms. Bush and Dr. Ballinger will address the implementation of PerfO assessments in multinational clinical trials, with a focus on the unique considerations related to task-based assessments and the special populations often involved; and Dr. Campbell will provide her perspective on regulatory expectations regarding fit-for-purpose PerfO assessments in the evaluation of clinical benefit. A moderated discussion with polling will follow to engage the audience in discussing implementation challenges and good practices.
Discussion Leaders
Sonya Eremenco, MA
Critical Path Institute, Tucson, AZ, USA
Discussants
Rachel Ballinger, PhD, BSc
ICON plc, Reading, RDG, United Kingdom
Rachel is a Principal in the Patient Centred Outcomes group at ICON. Since joining ICON in 2011 she has worked as a Project Director and Scientific Advisor, to undertake and support multiple studies. This has spanned various disease areas and methods, including in paediatrics, rare diseases, and oncology. She has also undertaken a multi-site evaluation study in performance outcome assessments and has presented on this, as well as considerations for use of wearables and sensors in outcome assessments.
Rachel has a PhD in Medical Sociology. She is well versed with all stages of the research process and enjoys collaborating with people from varied backgrounds and disciplines.
Bill Byrom, B.Sc., Ph.D.
Signant Health, Nottingham, NTT, United Kingdom
Bill serves as Vice President at Signant Health, a global company providing technology solutions for pharmaceutical clinical trials. He has worked in the Pharmaceutical industry for 30+ years and is a recognised industry leader in eClinical product strategy, electronic clinical outcome assessments (eCOA), and decentralized clinical trials. Bill is an experienced scientific expert and the author of over 70 publications, and two industry textbooks on electronic patient-reported outcomes (ePRO). His recent scientific work includes the use of wearable and sensor technology, and bring-your-own-device (BYOD) eCOA in clinical trials. Bill serves on two industry expert task forces within ISPOR, is a member of the scientific leadership board of the Digital Medicine Society (DiMe), and a core contributor to the US Critical Path Institute’s eCOA Consortium. Bill is a senior visiting professor within Nottingham Trent University's Medical Engineering Design Research Group.
Michelle Campbell, PhD
US Food and Drug Administration, Silver Spring, MD, USA
Dr. Michelle Campbell is the Associate Director for Stakeholder Engagement and Clinical Outcomes in the Office of Neuroscience, Office of New Drugs (OND) in FDA’s Center for Drug Evaluation and Research. Dr. Campbell joined the FDA in 2014 and previously was a reviewer on the Clinical Outcome Assessments (COA) Staff and Scientific Coordinator of the COA Qualification Program in OND. Dr. Campbell’s focus is in patient-focused drug development and the use of patient experience data in the regulatory setting. Prior to joining FDA, Dr. Campbell spent more than 10 years conducting research in the academic-clinical setting, including five years in a neurology and developmental medicine department. Dr. Campbell earned her BA in Biology from the College of Notre Dame, her MS in Health Science from Towson University, and her PhD in Pharmaceutical Health Services Research from the University of Maryland School of Pharmacy.
Horizon Europe Project Ascertain: Supporting a Sustainable and Transparent Legal EU HTA Framework, Accessibility of Innovative Technologies, and Health Equity
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Level: Intermediate
PURPOSE: To contribute to a sustainable and transparent legal EU HTA framework.
DESCRIPTION: The affordability of newly approved innovative health technologies (IHTs) poses a challenge for many health systems in the European Union (EU). To ensure quick patient access to these technologies, health care payers and pharmaceutical companies have explored various ways of defining payment for new products. However, the negotiation process between companies and individual countries has resulted in significant price discrepancies and unequal patient access. To address this issue, the EUnetHTA 2021 initiative has made progress in jointly assessing the clinical aspects of IHTs starting from 2025. Nonetheless, Member States still retain the responsibility of evaluating the added value of these technologies to their respective health systems. To establish a sustainable and transparent legal EU Health Technology Assessment (HTA) framework, the ASCERTAIN project aims to develop new models for pricing, cost-effectiveness, and reimbursement.
In this workshop, Mirjana Huic will provide an overview of the EU HTA regulation, its main objectives, key principles, governance structure, areas of joint work, and the role ASCERTAIN can play in supporting it. Isabelle Durand-Zaleski will then discuss the differences between countries using price and reimbursement as negotiation variables, along with the use of clinical effectiveness data for value-based purchasing. Nicolas Xander will elaborate on the development of new models within the ASCERTAIN project and addressing legal and sustainability issues. The workshop will conclude with an interactive question and answer session aimed at fostering cooperation between Member States and gathering input for the ASCERTAIN project. Policy makers, healthcare professionals, patients, academia, and health technology developers are encouraged to attend. The speakers will engage the audience by soliciting public comments and conducting real-time polling to gather immediate feedback and insights. Active participation is expected to gather diverse perspectives and experiences in expanding the HTA framework.
Discussion Leaders
Carin Uyl-De Groot, Prof Dr.
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Carin Uyl-de Groot (1966) is Professor of Health Technology Assessment (HTA) at the Erasmus University Rotterdam, the Netherlands. Until 2022,she was head of the HTA section of the Erasmus School of Health Policy&Management. She is director of the institute for Medical Technology Assessment (iMTA b.v.) since 2000. Her research activities are mainly focused on cost-effectiveness and outcomes studies in cancer. In 2018, she was chair of the public health and health economics track of the ESMO 2018 conference. Her papers in Nature Reviews (2018) about a new pricing model for (cancer) drugs and in Cancers (2020) about access to new cancer drugs across Europe, have attracted a lot of attention. Further, she is principal investigator of the Horizon Europe project ASCERTAIN.
Discussants
Isabelle Durand Zaleski, MD, PhD
ECEVE UMR 1123, CRETEIL, France
Isabelle Durand-Zaleski is a medical doctor, Professor in Public Health.
She carried out her PhD research in economics and management at Paris IX
University. She holds a Masters in Public Health from Harvard University and a
diploma from the Political Study Institute of Paris (Institut d’Etudes Politiques
de Paris). She has been the head of the Evaluation Department in the National
Health Authority. She is currently the head of the Paris Health Economics and
Health Services Research Unit in Paris.
She is author of 370 peer reviewed and Medline listed articles on health economics and health policy.
Mirjana Huic, MD, MSc, PhD
HTA/EBM Center, Zagreb, Croatia
Nicolas Xander, MSc
Erasmus University Rotterdam, Rotterdam, Netherlands
Nicolas Xander is a PhD candidate at Erasmus School of Health Policy and Management (ESHPM), Erasmus University Rotterdam. His background includes a degree in law (with several years of working experience as a lawyer and in state administration), as well as a master's degree in health economics, policy and law.
Mr Xander's research involves the development of pricing and cost-effectiveness models to improve patients' access to innovative health technologies, and the assessment of the clinical value of such technologies tested in single-arm trials.
Case Studies
Breakouts: IP, WS, & OBS
Challenges and Opportunities of Real-World Evidence in Emerging Countries
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Level: Intermediate
ISSUE: Real-world evidence (RWE) has emerged as an important tool for healthcare decision-making, including health technology assessment (HTA) and reimbursement of new and innovative treatments. This issue panel aims to explore the challenges and opportunities of RWE in emerging countries and discuss potential solutions to bridge the gap between RWE and healthcare decision-making.
OVERVIEW: The panel will bring together three experts with diverse backgrounds and experiences to discuss the current state of RWE in emerging countries, identify challenges and opportunities, and propose strategies to improve RWE generation and utilization. The panelists' perspectives will be as follows:
The importance of RWE for healthcare decision-making in emerging countries, including the role of RWE in supporting evidence-based healthcare policies and improving access to innovative treatments. The challenges of implementing RWE in emerging countries, including issues related to data quality, availability, and standardization, as well as regulatory barriers and cultural differences. The homework for decision-makers to implement RWE in emerging countries, including strategies to improve RWE generation and utilization in different healthcare systems and settings. The panel discussion will provide insights into the potential of RWE for improving healthcare decision-making in emerging countries and identify areas for future research and collaboration. The panelists will offer recommendations for policymakers, regulators, and healthcare stakeholders to promote the use of RWE in emerging countries, as well as strategies to overcome the challenges of RWE generation and utilization in these settings. This issue panel will provide a platform for stakeholders to share their experiences, discuss challenges and opportunities, and propose solutions to improve RWE generation and utilization in emerging countries. The panel discussion will contribute to the advancement of RWE as a valuable tool for healthcare decision-making in emerging countries, and help to ensure that all patients have access to safe, effective, and affordable healthcare.
Moderators
Birol Tibet, BA, MA, MSc, MD
Istanbul University, Istanbul, Turkey
Dr. Birol Tibet is a medical doctor and health economist specialized in HTA and public health who holds multiple degrees and has extensive experience in healthcare policy and management. His contributions to develop infrastructure for and promote real-world evidence and HTA at the Turkish MoH and NIH have been recognized through awards and leadership roles in international health organizations. Currently a lecturer at Near East University, he also co-leads ECONiX Research, providing consulting services in HEOR and market access strategies across Europe, North Africa, the Middle East, and Western Asia.
Panelists
Gihan Elsisi, Phd
The American University in Cairo, cairo, C, Egypt
Gihan Hamdy Elsisi, Msc, PhD is Assistant Prof, American University in Cairo. She is the founder and former Head of Pharmacoeconomic Unit at the Egyptian drug Authority (EDA), Egyptian Ministry of Health and consultant at USAID and WHO EMRO. Elsisi received PhD in Pharmacoeconomics /pharmaceutical sciences from Ain Shams University and certified in Health Economics (HE) and Outcomes Research, University of Washington. Through the EDA, Elsisi was able to successfully incorporate Pharmacoeconomic and Outcomes Research into the coverage decisions of drugs in Egypt and build HE capabilities in different Middle East countries. On international level, she is the Health Science Policy Council Advisor, at International Society for Pharmacoeconomic and Outcomes Research (ISPOR). She is the principal author of both Health Care Systems Roadmap for Pharmaceutical pricing and reimbursement and the Pharmacoeconomic Guidelines for Egypt, ISPOR. She is an executive member in HE Diploma Committee, American University in Cairo. Elsisi represented Egypt in the First and Second Middle East Africa Pricing & Reimbursement Future Trends Workshop in Muscat 2012 and in Dubai 2013. She has several publications in HE and also contributed to a number of clinical research and surveys. She is the treasurer of ISPOR Egypt Chapter. She is a special guest speaker at many international companies for achieving market access and maximizing commercial performance in North Africa & Middle East.
Guvenc Kockaya, MD, MSc, PhD
ECONiX Research, Samsun, 55, Turkey
Dr. Guvenc Kockaya is a medical doctor and health economist. He has a career of more than 15 years in market access & health economics with government, academia, and private industry experience. He is the editor of the books titled as “Pharmaceutical Market Access in Emerging Markets” and “Pharmaceutical Market Access in Developed Markets” which are still in the Top 100 Best Sellers (International) in Amazon under “Pharmaceutical & Biotechnology Industry (Kindle Store)”. He is the founder of ECONiX which is an internal company that gives tailor-made consultancy services including but not limited to market access, health economics & outcome research, medical affairs, and business development for government and academic institutes, pharmaceuticals, medical devices, and healthcare services companies in Eastern Europe, Middle East, North Africa, and Western Asia countries with offices in Estonia, Tunisia, and Turkey.
Mondher Toumi, MD, PhD, MSc
Aix Marseille University, Marseille, France
Professor Mondher Toumi is an MD by training and holds two MSc in Biostatistics, and in Biological Sciences (option pharmacology) and a PhD in Economic Sciences. He is a professor of Public Health at Aix-Marseille University. After working for 12 years as a research manager in the Department of Pharmacology at the University of Marseille, he joined the Public Health Department in 1993. In 1995, he entered the pharmaceutical industry and worked there for 13 years.
Mondher Toumi was appointed global vice president at Lundbeck A/S in charge of health economics, outcome research, pricing, market access, epidemiology, risk management, governmental affairs, and competitive intelligence. In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in strategic decision-making.
In February 2009, he was appointed professor at Lyon I University in the Department of Decision Sciences and Health Policies. He launched the first European University Diploma of Market Access (EMAUD) an international course already followed by more than 500 students. Additionally, he recently created the Market Access Society to promote research and scientific activities around market access, public health and health economic assessment. He is chief editor of the Journal of Market Access and Health Policy (JMAHP).
Since September 2014, he joined the research unit EA3279 of the Public Health Department, at Aix-Marseille University (France) as Full Professor. Mondher Toumi is also visiting professor at Beijing University (Third Hospital).
In June 2022 Mondher Toumi founded InovIntell an international venture dedicated to AI in life sciences.
He is a recognized expert in health economics and an authority on market access and risk management. He published more than 200 scientific publications and authored or co-authored several books predominantly in the fields of market access and health economics.
Embedding Environmental Sustainability in Technology Value Assessment: Challenges and Opportunities
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Level: Introductory
ISSUE: Combating climate change is a global challenge, affecting all stakeholders along the health value chain – most importantly patients. To unlock the full value of climate action by the biopharma industry, environmental sustainability criteria need to be implemented consistently in clinical development as well as regulatory and health technology assessments. Following questions arise:
How do regulators/policy-makers measure and value environmental sustainability and its impact? How to embed environmental impact in health benefit assessment and economic evaluation? How to incentivise corporate climate action? The objective of this session is to voice different perspectives on the potential value of environmental sustainability and inclusion in the design of pharmaceutical products and their value assessment. <
Jens Grueger > will introduce the concept of environmental sustainability in the HTA and conduct an audience poll on current understanding and importance of the topic.
<Sean Sullivan> will argue from his viewpoint for the inclusion of environmental sustainability as a component of value frameworks.
<Meindert Boysen> will discuss how an organization like NICE considers environmental sustainability in their assessments.
< Alessandra Madoni > will provide an industry perspective on efforts to include environmental sustainability in product design and what incentives are required to embed this more broadly. <
Jens Grueger > will then invite audience participation and moderate a panel discussion and Q&A.
OVERVIEW:
10’ topic introduction highlighting recent issues and policies10 ' health economic perspective of including environmental sustainability as a component of value frameworks10’ challenges and perspectives on including environmental sustainability in HTA assessments10’ real-life industry experience on commitment to environmental protection and impact on product design20’ panel discussion and Q&A
Moderators
Jens Grueger, PhD
Boston Consulting Group, Zurich, Switzerland
Jens Grueger is a Director and Partner at the Boston Consulting Group based in Zurich Switzerland and a Fellow of the BCG Henderson Institute. He provides expert advice to companies in the life science industries on pricing, access and affordability. Since July 2019, he is an Affiliate Professor of Health Economics at the CHOICE Institute at the University of Washington School of Pharmacy in Seattle, USA. He was the 2020-2021 ISPOR President, the leading professional society for health economics and outcomes research.
Prior to this, Jens had a distinguished career in the pharmaceutical industry. From 2011-2019, he was the Senior Vice President and Head of Global Access for F. Hoffmann-La Roche based in Basel, Switzerland. Under his leadership, Roche created rapid, broad and sustainable patient access for 12 new medicines and expanded access to essential cancer medicines in lower- and middle-income countries from 29 to 60%. Before this Jens was the Vice President and Head of Global Market Access Primary Care at Pfizer, based in New York and London (2009-2011), Head of Global Pricing & Health Economics at Novartis Pharma in Basel, Switzerland (1999-2009) and Director of Health Economics at the German affiliate of SmithKline Beecham Pharma in Munich (1994-1997).
Panelists
Meindert Boysen, PharmD, MSc
Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Meindert is responsible at NICE for international partnership working, developing NICE’s role in international fora and representing NICE at key international meetings, further sustaining and developing NICE’s international reputation. Until recently, Meindert was the director at NICE responsible for delivery of the programmes that together form the centre for health technology evaluation. Including the technology appraisals, highly specialised technologies, medical technologies evaluation, diagnostic assessment and interventional procedures programmes. After qualifying as a pharmacist from Utrecht University, he worked in a hospital in the Netherlands, and held positions in health outcomes and sales in the pharmaceutical industry. Meindert completed the MSc in Health Policy Planning and Financing at the London School of Hygiene and Tropical Medicine and the London School of Economics & Political Sciences and briefly worked for the King’s Fund before starting at NICE in 2004. Meindert was a member of the Board of NICE and a director of the board of the International Society for Pharmacoeconomics and Outcomes Research (2017-2020).
Alessandra Madoni, MSc, Pharm.D
Chiesi Farmaceutici Spa, PARMA, PR, Italy
Alessandra, the Head of Global Value and Access Respiratory at Chiesi Farmaceutici, a B Corp and certified benefit corporation in Italy and the US, is responsible for managing the company’s respiratory portfolio. She supports project prioritization, new products access strategies, and provides input to clinical development. She is dedicated to improving patient access to Chiesi solutions and assessing new opportunities. Alessandra leads sustainability projects and promotes a broader approach to product assessment. She represents Chiesi in the EFPIA – HTA Working Group a. Additionally, she is an Adjunct Professor of Health Economics at the University of Parma, Italy.
Sean Sullivan, BScPharm, PhD
The CHOICE Institute School of Pharmacy, University of Washington, Seattle, WA, USA
Sean D. Sullivan, BScPharm, MSc, PhD, is Professor, School of Pharmacy, University of Washington and Visiting Professor, London School of Economics. He holds a joint appointment in the School of Public Health. He holds adjunct appointments in the School of Medicine and the Public Health Sciences Division at the Fred Hutchinson Cancer Research Center.
He completed training in pharmacy at Oregon State University in 1983, obtained a master’s degree at the University of Texas in 1986 and a PhD in health economics and policy at the University of California, Berkeley in 1992.
Dr. Sullivan was a member of the Medicare Evidence Development and Coverage Advisory Committee, a member of the Regence Blue Shield and Premera Blue Cross P/T Committee, and Chair of the Premera Blue Cross Value Assessment Committee. He was awarded the 2014 Stephen G. Avey Lifetime Achievement Award from the Academy of Managed Care Pharmacy (AMCP), the 2015 APhA Academy of Pharmaceutical Research Sciences (APRS) Research Achievement Award, and was elected to the National Academy of Medicine in 2020.
Measuring Health Related Quality of Life in Pediatric Life Limiting Progressive Diseases: Whose Perspective Are We Measuring?
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Level: Intermediate
ISSUE: There are methodological challenges in how we measure Health Related Quality of Life in paediatric progressive life-limiting conditions. There are inconsistent approaches to whose perspective is measured: children, proxy or population. Can we reach a consensus on how to capture HRQoL in paediatric progressive life-limiting conditions? Can we account for the inevitable adjustments made by these children over their lifetime? Should we incorporate the value of hope for untreatable life limiting conditions?
OVERVIEW: This panel will discuss different approaches to including HRQoL in paediatric life-limiting conditions in HTA. Which perspective (proxy, patient or population) is most useful to decision-making, how to take into account the impact of disease progression over a lifetime and how best to interpret the results of HRQoL measures in clinical trials.
Josie Godfrey will chair the session and briefly present the challenges faced in incorporating HRQoL of paediatric life-limiting progressive conditions into HTAs. The panellists will take an international perspective and represent different stakeholder views. Ron Akehurst will take an HTA perspective, sharing views of the strengths and weaknesses of current HTA approaches to incorporating paediatric HRQoL into HTA. Fleur Chandler, a carer and health economist will present a perspective arguing for more consistency and pragmatism in measuring paediatric HRQoL and talk about the work of Project HERCULES in capturing what is important to patients and their families. Jill Carlton, an academic researching HRQoL and burden, will outline her recent work exploring common themes of importance in paediatric progressive life-limiting conditions and highlight emerging research recommendations with reference to differences noted between stakeholder groups. The panel will consider questions about the specific challenges of measuring HRQoL in progressive paediatric conditions and the role of collaboration between patient groups, academics, manufacturers and agencies.
Moderators
Josie Godfrey, MA
JG Zebra Consulting, Hammersmith, United Kingdom
Josie has worked in rare diseases and innovative therapies for 14 years. She currently runs a consultancy business specialising in strategic market access, policy and stakeholder engagement. She is the Strategic Director for Duchenne UK’s Project HERCULES, an award-winning global collaboration developing evidence and tools to support HTAs for new treatments for Duchenne Muscular Dystrophy. Josie is also co-founder and joint CEO of Realise Advocacy, which supports patient involvement in drug development and access processes. She previously led work at NICE to establish the Highly Specialised Technologies programme.
Panelists
Ron Akehurst, DSc, Hon MFPHM
Lumanity, Sheffield, South Yorkshire, United Kingdom
Ron is Chairman of Lumanity HEOR and an Emeritus Professor at the School of Health and Related Research (ScHARR), University of Sheffield, where he served as founder and Dean for 17 years.
He was a member of the first NICE Technology Appraisal Committee, and has continuously served on multiple NICE decision making committees since. He remains on the HST Committee at NICE and RDAG at NHS England, working in the Rare Disease space.
Ron has worked with governmental policy bodies and pharmaceutical companies over many years and various jurisdictions.
Jill Carlton, BMedSci, MMedSci, PhD
University of Sheffield, Sheffield, United Kingdom
Jill is a senior research fellow in at the School of Health and Related Research at the University of Sheffield, UK. Jill’s research interests lie in measuring and valuing health, primarily through the development of patient reported outcome and preference-based measures. She is a mixed methods researcher who has led projects across numerous health conditions. This includes children and carers, as well as in rare, progressive life-limiting conditions. Jill has been involved in many projects which have had active stakeholder engagement, including developing a framework for fully incorporating public involvement (PI) into patient-reported outcome measures (PROMs).
Fleur Chandler, MSc
Sanofi and Patient Advisory Board Lead, Duchenne UK, Reading, Berkshire, Great Britain
Fleur is Head of Market Access UK & Ireland at Sanofi. Fleur is a health economist with a wealth of outcomes research and health technology assessment experience, with over 30 years leading both global and local teams in the pharmaceutical industry. Fleur is especially driven by rare disease, a parent of a teenager with Duchenne Muscular Dystrophy, she is on the advisory board of patient organisation Duchenne UK. Bringing her professional and personal experience together, she conceptualised and leads Project HERCULES, a global collaboration generating gold standard disease level evidence for HTA in Duchenne.
Gender and Health Equity in Health Care Decision-Making: How Women's Issues Continue to Be Neglected
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Level: Introductory
ISSUE: Marked health inequalities related to gender persist worldwide as recent advances in health outcomes (such as life expectancy, living with disability, dementia) have not translated equally for men and women. Little consideration has been given to the changing health needs and risks of women throughout their lifetime. Recent attention on the impact of health inequalities in decision-making, including health technology assessment (HTA) and health policy, has focused exclusively on race and ethnicity, leaving gender and socioeconomic disparities in the background.
The panel will debate why women may have been traditionally overlooked in HTA and health policy considerations, and the need to generate gender-specific evidence to identify disparities and enhance equitable decision-making.
OVERVIEW: Stark health inequalities between women and men are well documented. Gender discrimination, lack of education and domestic violence have exacerbated health inequalities for women. While progress has been made in HTA decision-making to address health inequalities among marginalised groups, the focus has mainly been on race/ethnicity, while women and socioeconomics’ issues have largely been ignored.
The moderator will introduce this topic by presenting the evidence around gender health inequalities and the urgent need to reshape decision-making to include health outcomes specific to women (10 minutes). Each panelist will present a different perspective (women’s advocate, industry, philantropic) why HTA processes and health policy have fallen behind in considering the implementation of gender health equity (10 min each). The panel will debate whether gender-responsive HTA reform needs to start by challenging gender stereotypes in healthcare or if urgent action is needed given methodological advances, availability of patient-level data in routine clinical practice and expanded economic models. The session will conclude with an interactive discussion with the audience and the use of online polling questions (10 minutes). Stakeholders from regulatory, HTA, industry, payers and research organisations will benefit from this session.
Moderators
Grammati Sarri, PhD, MSc, DiDS
Cytel, London, United Kingdom
Grammati has 15+ years’ experience in epidemiology studies, evidence synthesis and decision making from her prior roles in academia, health technology assessment (NICE, UK) and private health consultancies. Grammati has strong methodological experience in generating impactful evidence from different sources (clinical trials, real-world evidence, economic studies), clearly communicating findings in an impactful way and using complex analytical methods for health care-decision making. Grammati currently holds the position of Chair of the ISPE comparative effectiveness research special interest group and is a topic co-editor for the Journal of Frontiers in Pharmacology. Grammati has numerous presentations (both as a moderator and panelist) in previous ISPOR conferences. For the particular topic, she has published a white paper https://pubmed.ncbi.nlm.nih.gov/35784688/ which was widely communicated and presented initial results of an ISPE funded project on unraveling health disparities in COVID-19 treatments at an issue panel at ISPOR US 2023.
Panelists
Ru-fong Cheng, MD, FACOG
Bill and Melinda Gates Foundation, Skillman, NJ, USA
Ru-fong Joanne Cheng, MD, FACOG (she/her/hers)
Director, Women’s Health Innovations
Dr. Ru Cheng is a women’s health physician-researcher, product developer, and health equity advocate. She currently leads a team at the Bill & Melinda Gates Foundation that supports the development and adoption of high-quality health products that address the needs of women and girls in low- and middle-income countries. The Women’s Health Innovations team also serves as a unifying voice on women’s health, collaborating with other foundation teams and external partners to direct more funding and attention to R&D for women's health.
Ru enjoys tackling challenges by combining her clinical insights from academic medicine with her product development experience from over 15 years in the biopharmaceutical industry in the clinical development of drugs and devices for women’s health at Johnson & Johnson, Bayer, and Pfizer.
Jay Jhutti-Johal, PhD
University of Birmingham, Birmingham, United Kingdom
Montse Soriano Gabarro, MD, MSc, FISPE
Bayer, Berlin, BE, Germany
Montse Soriano-Gabarro, MD, MSc, FISPE
Physician, preventive medicine specialist and epidemiologist with more than 20 years of experience working in healthcare, government and the pharmaceutical industry. Broad experience on the use of observational real world data sources, study designs and technologies applied to communicable, non communicable diseases, vaccine and drug development and evaluation. Currently Head of Partnerships, Policy and Integrated Evidence Generation (IEG) Office at Bayer AG, Germany. Strong interest on best application of innovative epidemiological data sources and methods to support regulatory and health policy decisions. Bayer Distinguished Science Fellow, Fellow of the International Society for Pharmacoepidemiology (ISPE) and co-chair of ISPE's Real World Evidence Collaborative Special Interest Group (SIG).
17:30 - 18:00
ISPOR HEOR Awards Celebration Presentation
Join us as we present the awards to the 2023 HEOR Awards Honorees, who have made outstanding contributions and demonstrated exceptional leadership in the field. ISPOR Scientific Awards are designed to foster and recognize excellence and outstanding achievement in HEOR, and its Leadership Awards recognize excellence and outstanding leadership in the field.
18:00 - 18:30
ISPOR HEOR Awards Celebration Reception
Join us for a reception at the ISPOR Booth to celebrate the 2023 HEOR Awards Honorees who have made outstanding contributions and demonstrated exceptional leadership in the field. ISPOR Scientific Awards are designed to foster and recognize excellence and outstanding achievement in HEOR, and its Leadership Awards recognize excellence and outstanding leadership in the field.
18:00 - 19:00
Welcome Reception
Taking place in the Exhibit Hall
18:30 - 19:00
Meet ISPOR's New CEO and Engage With ISPOR Leaders
Join us for a reception at the ISPOR Booth to welcome ISPOR's new CEO, Rob Abbott, and meet members of the board of directors, as well as other ISPOR senior leaders!
Tue 14 Nov
7:00 - 17:00
Registration Hours
7:30 - 8:30
Morning Coffee Service
8:30 - 9:45
Plenary Session
The New Pharma Legislation Proposal: The Good, the Bad or the......
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After long and difficult negotiations, the EC adopted a proposal for the revision of the EU pharmaceutical legislation at the last possible moment to allow a decision on the implementation during the current legislative period. This proposal is the outcome of a process initiated in November 2020 aiming to create a future proof regulatory framework and at supporting the industry in promoting research and technologies that reach patients to fulfill their therapeutic needs while addressing market failures.
4 core pillars were identified in 2020
• Ensuring access to affordable medicines for patients, and addressing unmet medical needs (in the areas of antimicrobial resistance and rare diseases, for example)
• Supporting competitiveness, innovation and sustainability of the EU’s pharmaceutical industry and the development of high quality, safe, effective and greener medicines
• Enhancing crisis preparedness and response mechanisms, diversified and secure supply chains, addressing medicines shortages
• Ensuring a strong EU voice in the world, by promoting a high level of quality, efficacy and safety standards.
This panel will discuss whether these original intentions are still sufficiently reflected in the latest proposal after several rounds of consultations of different Stakeholder groups and revisions. The group will also discuss how and where HEOR might help with identified shortcomings or omissions in the new proposal as identified by the panel.
Moderators
Anja Schiel, PhD
Norwegian Medicines Agency (NoMA), Oslo, 02, Norway
Anja Schiel works as Special Adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects at the Norwegian Medicines Agency. She has been Chair of EMA’s Biostatistics Working Party 2017 – 2019 and from 2019 – 2022 she was Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member at the SAWP and is member of the new Methodology Working Party (MWP) recently established at EMA.
She has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21 with a particular focus on parallel EMA-HTA scientific advices.
Speakers
Gloria Ghequiere, MEUS
Belgium Federal Government, Brussels, Belgium
Neil Grubert, MA
Independent, Basildon, United Kingdom
Denis Lacombe, MD
European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium
Denis Lacombe graduated with his MD from the University of Marseilles (France) in 1988 and obtained a Master Post-Doctoral Fellowship at The Roswell Park Cancer Institute (Buffalo, NY USA) for research in pharmacology and pharmacokinetics from 1989 to 1991. From 1991 to 1993, he worked as a Clinical Research Advisor in charge of the development of a new drug in oncology in the pharmaceutical industry. Dr Lacombe joined the EORTC in 1993 as a research fellow and quickly became a very active and productive Clinical Research Physician involved in the conduct of clinical research from protocol development through publication for a number of oncology indications from phase I to phase III. Dr Lacombe contributed to the strategic evolution of the EORTC pan-European clinical and translational research infrastructure as well as developing partnerships with multiple stakeholders. Dr Lacombe rose to the position of Director EORTC Headquarters in 2010, was appointed Director General in April 2015 and Chief Executive Officer in June 2021. In his current position, Denis Lacombe leads all activities promoting the EORTC as a major European organization in Cancer Clinical and Translational Research, including scientific activities, public relations and mediumterm strategies as defined by the EORTC Board as well as internal and external communications. Dr Lacombe is the author of well over 150 peer reviewed publications and communications.
Yannis Natsis, MA, BA
European Social Insurance Platform (ESIP), Brussels, Belgium
Yannis Natsis is the Director of the European Social Insurance Platform (ESIP), the umbrella organisation bringing together 45 national statutory social security institutions from 18 countries. ESIP is the voice of social protection and security in Europe or as Yannis puts it one of Europe’s truest treasures.
He has more than 10 years of experience in EU advocacy and policymaking. Prior to joining ESIP in February 2022, he led the advocacy for better and affordable medicines at the European Public Health Alliance (EPHA). In May 2019, he was appointed by EU Member States to the Management Board of the European Medicines Agency (EMA), a position he held until December 2021. Additionally, Yannis has been a Board member of the European Health Forum Gastein (EHFG), the leading EU health policy platform since 2018.
Yannis previously worked for the TransAtlantic Consumer Dialogue (TACD) focusing on health and pharmaceutical policies. From 2006-2010, he was an investigative reporter for Greece's award-winning TV news programme "Fakeli" and a contributor to one of Greece’s most respected dailies “Kathimerini”.
He has a Master's degree in International Conflict Analysis from the University of Kent, UK and a Bachelor's degree in European Studies from Pantion University of Social and Political Sciences, Athens, Greece. A Greek national, he is fluent in Greek, English and French.
Johan Pontén, MA
Medicine Evaluation Committee (MEDEV), Stockholm, Sweden
Johan Pontén is Senior Manager of International Affairs at The Dental and Pharmaceutical Benefits Agency in Sweden since 2014. He is since 2021 co-chair of the Medical Evaluation Committee, MEDEV, a network of 22 national authorities from 18 Member States and Norway bringing together all the relevant institutions (national HTA agencies and social health insurers-payers) responsible for the assessment, pricing and reimbursement of medicines in Europe. He has also supported the FINOSE collaboration between Nordic HTA agencies from the start in 2017. He has had a career in the Swedish Public Service and holds a Master in Pedagogic Leadership.
9:30 - 19:00
Exhibit Hall Open
9:45 - 10:15
Coffee Break
Located in the Exhibit Hall
10:15 - 11:00
Let's Talk HEOR Workforce Diversity
Join us for refreshments and discussion about workforce diversity in HEOR. This event presents a unique opportunity for participants to partake in an informal discussion with the members of the ISPOR HEOR Workforce Diversity Cross-Council Working Group, focusing on the important topic of HEOR workforce diversity and the role that ISPOR plays in championing these efforts. During this interactive booth event, attendees can connect with Initiative leaders and learn how to get involved.
10:15 - 11:15
Podium Sessions
Adherence to Treatment, Patients' Preferences, and Drug Use Across Multiple Diseases
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This podium explores the importance of better considering adherence to treatment across several diseases. It explores the impact of adherence to lenalidomide oral maintenance therapy on patients' quality of life in Multiple Myeloma, how non-adherence among individuals can be related to their level of depression, to what extend patients with Parkinson disease can face treatment discontinuation, and how "truthful" self-reported adherence contributes to explain heterogeneity in treatment preferences.
Moderator
Jorien Veldwijk, PhD
Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, South-Holland, Netherlands
Assistant professor at the Erasmus School of Health Policy & Management and member of the Erasmus Choice modelling Centre. Research focuss on preference elicitation within a broad range of (public) health care topics as well as methodological issues.
P26: Drug Utilization, Adherence, and Persistence in Parkinson's Disease Patients in a Real-World Setting in Quebec, Canada
10:30AM - 10:45AM
Baribeau V1 , Mohammed S2 , Awan A3 , Parison D2 , Lachaine J 1 1 PeriPharm Inc, Montreal, QC, Canada, 2 AbbVie, Montreal, QC, Canada, 3 AbbVie Corporation, Saint-Laurent, QC, Canada
OBJECTIVES: Parkinson’s Disease (PD) is a progressive neurodegenerative disease that affects the control of movement, with a variety of symptoms such as rigidity and tremor. There is no cure for PD and the goal of treatment is to relieve symptoms. The objective of this project was to analyze drug utilization, adherence and persistence in a real-world setting, of patients with a diagnosis of PD in Quebec, Canada.
METHODS: Using data from the Régie de l’Assurance Maladie du Québec (RAMQ) database for the period from January 2010 to December 2018, a sample of PD patients was selected. Patients had to have at least one diagnosis code for PD (ICD-9 code 332.0 or ICD-10 code G20.x) and received a PD medication (levodopa, dopamine agonist, catechol-o-methyl transferase inhibitor, monoamine-oxidase-B inhibitor and/or anticholinergic drug) at least once. Treatment utilization was measured during the follow-up (the end of the RAMQ coverage). Adherence to PD treatments was estimated by calculating the medication possession ratio (MPR) that corresponds to the total number of days’ supply of medication dispensed divided by the duration of treatment. Persistence rates were measured using a Kaplan-Meier analysis.
RESULTS: A total of 1,212 patients were analyzed. The average age was 74.3 years and 56.8% were male. The drug most often used was levodopa/carbidopa (89.8%), followed by pramipexole (17.2%), and rasagiline (14.5%). A higher proportion of patients had only used 1 treatment (49.8%). Average adherences were high for all classes of treatment (MPR≥85%) and were consistent over time, i.e. from 3 months to 24 months of treatment. The discontinuation rates at 24 months for dopamine agonists, catechol-o-methyl transferase inhibitors, monoamine-oxidase-B inhibitors, and anticholinergic drugs were approximately 50% (47.6% to 49.5%), but the levodopa class discontinuation rate was lower (20.4%).
CONCLUSIONS: While on treatment, patients were adherent. However, there were high rates of discontinuation.
P27: How Untruthfulness in Self-Reported Medication Adherence Influences Treatment Preferences: A Discrete Choice Experiment
10:45AM - 11:00AM
Oedingen C 1 , van Gestel R2 , Huls S1 , de Bekker-Grob E1 , Veldwijk J1 1 Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, South-Holland, Netherlands, 2 Erasmus School of Economics, Erasmus University Rotterdam, Rotterdam, South-Holland, Netherlands
OBJECTIVES: Although self-reported questionnaires are commonly used to measure medication adherence, self-reported adherence may be influenced by socially desirable answers resulting in untruthful reporting. Such untruthful reporting can have serious consequences for reporting on treatment (cost)effectiveness. This study investigated how to induce truthful self-reported adherence and how this can explain heterogeneity in treatment preferences.
METHODS: A survey measuring self-reported medication adherence was administered immediately after a discrete choice experiment regarding preferences for Multiple Sclerosis (MS) treatments. Data was collected among MS patients in the Netherlands, France and the United Kingdom. Half of the sample was randomized to receive choice-matching which was used to induce truthful responses towards questions on adherence. Choice-matching (financially) incentivizes respondents depending on individuals’ predictions about others’ responses in the same sample, and the choice predictions of others with the same adherence score. Descriptive statistics and mixed logit models with interaction effects were conducted to determine the influence of choice-matching on self-reported adherence and to assess whether truthful adherence explains preference heterogeneity.
RESULTS: The sample comprised 758 MS-patients (mean age 40.8 years, 67% female). On average, respondents reported a higher adherence if they only received the adherence questions compared to respondents also receiving choice-matching (always adherent: 46.6% vs. 39.3%). The mixed logit model showed statistically significant interaction effects between the self-reported adherence and preferences for treatment modality. Only in the choice-matching group, higher adherence resulted in lower utility scores for implants compared to injections or pills (p=0.01).
CONCLUSIONS: Choice-matching encouraged respondents to report their true medication adherence. Linking truthful adherence to preferences allows for a better understanding of preference heterogeneity, better (cost)effectiveness assessments of treatments and can help patients and physicians with making a shared treatment decision that fits the patients’ true preferences.
P25: Association between Adherence to Lenalidomide and Patient-Reported Outcomes in Patients with Multiple Myeloma: A Systematic Literature Search
10:15AM - 10:30AM
Ramasamy K1 , von Lilienfeld-Toal M2 , Maisel C3 , Gustavsson B 4 , Bäck K5 , Glasmacher A6 , Leleu X7 1 Oxford University Hospitals NHS Trust, Oxford, Oxfordshire, UK, 2 Universitätsklinikum Jena, Jena, Thuringia, Germany, 3 Baylor University Medical Center, Dallas, TX, USA, 4 Nanexa AB, Uppsala, Sweden, 5 Nanexa AB, Uppsala, Uppland, Sweden, 6 University of Bonn, Bonn, North Rhine-Westphalia, Germany, 7 CHU Poitiers, Poitiers, Nouvelle-Aquitaine, France
OBJECTIVES: Lenalidomide, a molecular-targeted agent that binds to cereblon, is active in multiple myeloma (MM), the second most common haematological malignancy. Continuous exposure is associated with efficacy; however, the impact of adherence to lenalidomide oral maintenance therapy on quality of life is unclear. We assessed the available literature to assess the relationship between adherence and patient-reported outcomes (PROs) in lenalidomide-treated MM patients.
METHODS: We conducted a systematic literature search for articles published before 31Mar2023 assessing treatment compliance in patients with MM treated with lenalidomide using the PubMed and Embase databases. Data were assessed descriptively to determine the feasibility of a meta-analysis.
RESULTS: 321 articles were screened; 7 articles comprising 1,261 patients were eligible: 3 prospective real-world studies, 2 retrospective analyses, and 2 prospective clinical trials. Many studies included patients using lenalidomide in combination, most often with dexamethasone or ixazomib. Medication possession ratio (MPR) was most often used to quantify adherence. Where reported, adherence was quite high, with a median MPR 73–89.5% (n=2). In studies that used an MPR good-adherence cut-off of ≥80%, 3–38% of patients failed to meet this measure (n=3). However, lenalidomide therapy duration was not accounted for when interpreting MPR.
The most common PROs assessed were SATMED-Q
® , QLQ-C30, and QLQ-MY20. There was no clear association found by studies that tested correlations between adherence and PROs (n=4). One study found high treatment satisfaction was associated with good adherence. Pre-existing depression, adverse events, and less support from others were found to be associated with poor adherence (n=3).
CONCLUSIONS: A relevant proportion of MM patients treated with lenalidomide had an MPR of <80%; however, a definite conclusion was not drawn as the assessment of any relationship by means of a meta-analysis is confounded by variability in patient population, prescribing, and outcome measures used to assess both adherence and PROs.
P28: Medication Non-Adherence in Patients with Varying Levels of Depression Symptoms Measured Using the Medication Adherence Reasons Scale
11:00AM - 11:15AM
Unni E1 , Sternbach N2 , Costantino H3 , Gupta S 4 1 Touro College of Pharmacy, New York, NY, USA, 2 Cerner Enviza, Malvern, MO, USA, 3 Cerner Enviza, an Oracle Company, Kansas City, MO, USA, 4 Cerner Enviza, an Oracle Company, Flemington, NJ, USA
OBJECTIVES: Depression affects approximately 280 million people worldwide. Literature reports that the overall non-adherence to antidepressants immediately after the start of treatment was 13 to 55.7% and by six months was 52%. However, what is missing in the literature is the extent of non-adherence among individuals based on their level of depression. PHQ-9 score has been used to determine the level of depression as none to minimal, mild, moderate, moderately severe, and severe.
METHODS: Data was used from the 5EU 2022 National Health and Wellness Study (NHWS), a self-administered, annual, internet-based cross-sectional survey of adults in France, Germany, UK, Italy and Spain. Non-adherence was measured using the Medication Adherence Reasons Scale (MAR-Scale) which includes 19 reasons for non-adherence and one global item. NHWS participants who self-reported taking daily prescription medication(s) to treat depression responded to the MAR-Scale. Frequencies were used to identify the reasons for non-adherence.
RESULTS: Of the 4008 respondents (64.22% female; 48.42 mean age), 42.64% were non-adherent. Based on their level of depression, patients with moderate-severe depression were significantly more non-adherent than those with minimal or mild depression. Based on the 19 reasons, 72.27% of the none to minimal depression were adherent, but it decreased to 50.95% for those with severe depression. For all levels of depression, the major reasons were concerns about long term effects and potential side effects from the medicine, forgetfulness, and skipping the medicine to see if it is still needed. Those non-adherent with moderately severe depression missed the medicine an average of 3.04 days/week.
CONCLUSIONS: With ~50% of the patients with moderate-severe depression not taking medicines as prescribed due to concerns about the medicine, providers, payers, and pharmaceutical companies should develop educational interventions to improve adherence.
Impact of Regulatory Affairs on Access
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Moderator
Ka Keat Lim, MPharm, MSc, PhD
Kings' College London, London , LON, United Kingdom
Ka Keat Lim is a health economist currently working as a research fellow at King's College London (KCL), UK. At work, he works mainly on systematic reviews, longitudinal data analyses and economic evaluations, with particular interests on precision medicine, prevention and screening interventions. He is also the Co-Chair of Early Career Researchers & Teachers Network (ECRTN) at KCL School of Life Course & Population Sciences. Outside of work, he is a Member of the ISPOR New Professionals Committee, and Chair-Elect of ISPOR Precision Medicine & Advanced Therapy SIG. He enjoys reading non-fictions, listening to podcasts and strolling in the park during his free time.
P29: Conditional Marketing Authorization – Becoming Increasingly More Common But Increasingly Less Relevant
10:15AM - 10:30AM
Macaulay R 1 , Bear M2 1 PRECISIONadvisors, Edinburgh, UK, 2 Precision Advisors, London, LON, UK
OBJECTIVES: The European Medicines Agency (EMA) can grant a Conditional Medicines Authorization (CMA) for medicines that treat severe diseases based on less comprehensive clinical data than usual, pending the collection of more comprehensive data post-authorization. This research evaluates how the number of CMAs have evolved over time and their conversion to successful reimbursement.
METHODS: All publicly-available EMA CMAs were identified (01-JAN-2006–31-DEC-2022), alongside any corresponding NICE evaluation over the past 5 years (01-JAN-2018–29-JUN-2023) and key information was extracted.
RESULTS: 81 EMA CMAs were identified, representing 6.0% of all EMA medicine authorizations (range:0.9%[2009]–14.1%[2021]). There is a clear trend towards these becoming more common over time: CMAs represented over 10% of all EMA approvals every year since 2019, but was under 10% in every year before 2009. However, only a proportion of these have been converted to successful NICE reimbursement recommendations over the past 5 years, with 14% recommended, 14% optimized, 18% recommended through CDF, 9% not recommended, 9% terminated/discontinued, 18% ongoing, and 18% N/A (primarily COVID vaccines). This trend also appears to be substantially more negative over time, with 68% of NICE appraisals being ‘Recommended’/’Optimized’/’CDF’ prior to 2021 but the corresponding figure has fallen to 23% from 2021 onwards.
CONCLUSIONS: EMA CMAs are becoming an increasingly common route to market for new medicines, representing over 10% of new EMA approvals since 2019. We have shown that only a proportion of medicines translated this expedited marketing authorization into successful reimbursement outcomes in the UK, and this trend is becoming more negative over time. This is despite the UK being one of the few payer bodies with a dynamic reimbursement pathway that allows temporary reimbursement whilst additional data is collected (Cancer Drugs Fund and Innovative Medicines Fund). Further research can elucidate if this trend is mirrored across other major European markets.
P32: Implementation of Early Access Reform in France and Access to Innovation: Impact in Terms of Number of Drugs, Patients Treated, and Cost
11:00AM - 11:15AM
Martin T 1 , Rioufol C2 , Martelli N3 , Madelaine I4 , Borget I5 1 Paris Saclay University, Paris-Saclay, France, 2 Claude Bernard University, Lyon, Rhônes Alpes, France, 3 Université Paris Saclay, Orsay, FRANCE, France, 4 Saint-Louis Hospital, Paris, Ile de France, France, 5 Institut Gustave Roussy and Paris Saclay University, Villejuif, ile de France, France
OBJECTIVES: An ambitious reform of the Early Access (EA) process was set up in July 2021 in France, aiming to simplify procedures and accelerate access of innovative drugs. Two pathways now exist: authorization for early access (AEA) and authorization for compassionate use (ACU), instead of 6 under the former Authorization for use (ATU) system. The objective of this study was to describe the impact of the reform on the number of drugs in EA, number of patients treated and cost.
METHODS: This was an observational longitudinal retrospective study performed using the French Nationwide claims database (SNDS) data, which concerned patients treated with an EA drug at hospital between 2019 and 2022. Costs were evaluated in €2022 from the French National Health Insurance’s perspective. The impact of the reform was evaluated in terms of number of indications, number of patients treated and cost before and after the reform. A focus on oncology and onco-hematology drugs was done.
RESULTS: Between 2019 and 2022, the number of patients treated by an EA drug has increased from 22,627 to 58,829 patients (+ 160%), explained by a great increase concerning the AEA patients (from 5,516 to 39,224, +611%) and the number of AEA drugs available (from 26 to 120). Costs for EA treatments followed the same trend, with €803 million in 2022 compared to €369 million in 2019 (+118%) and a large share of AEA treatments (€608.64 million in 2022, 76%). Oncology and onco-hematology indications concentrate 59% of the drugs approved, 79% of the number of patients treated and 86% of expenditure over the period for AEA treatments.
CONCLUSIONS: Early access reform in France has allowed the rapid arrival of a significant number of therapeutic innovations over the past 18 months, as well as a great increase of the number of patients treated, mostly in oncology and onco-hematology.
P30: Do Novel Oncology Combination Therapies Have a Place Within the EU Legislative Framework? The Impact of Challenges for Novel Combination Therapies on Equality of Access Between European Patients
10:30AM - 10:45AM
Pistollato M 1 , Veale T2 , Ong SJ2 1 Charles River Associates, London, LON, UK, 2 Charles River Associates, Cambridge, CAM, UK
OBJECTIVES: Combination therapies have contributed to recent improvements in cancer care but encounter significant challenges impacting patient access. These challenges, and the potential to address them, vary considerably across European countries. Consequently, there is substantial inequality in treatment opportunities which we have documented through quantitative, evidence-based analysis. Additionally, the upcoming European legislative reforms (EU HTA Regulation, EU pharmaceutical legislation) have been considered; these could provide an opportunity to address challenges or further exacerbate existing access problems. This research aims to quantify the impact of access challenges faced by novel oncology combinations, and show the ongoing delays and sub-optimal availability of such therapies in selected European countries, to highlight the need for solutions.
METHODS: Two analyses were performed: overall availability of novel oncology combination therapies in England, France, Germany, Italy and Spain, and the average time taken for combination therapies to become available following EMA approval. Firstly, a database of all EMA-approved products between August 2015 – May 2023 was analyzed and filtered for non-generic/biosimilar products approved in oncology combination therapies. Data was then pulled from a third-party HTA/reimbursement database on reimbursement decision dates, recommendations, and EMA approval dates for each of the 33 novel combination therapies.
RESULTS: Novel oncology combination therapies are facing patient access delays and sub-optimal reimbursement: across EU4 and England, an average of 68% of EMA-approved novel oncology combination therapies are reimbursed, compared to 82% of all approved oncology products; combination therapies also take on average 40% longer to become available compared to all oncology products. Reimbursement also varies substantially between European countries: 32 oncology combination therapies approved between 2015-2023 are available in Germany, compared to only 14 in Spain.
CONCLUSIONS: Delayed patient access and sub-optimal reimbursement of novel oncology combination therapies are preventing treatment optimization and risk disincentivizing innovation/investment from the pharmaceutical industry towards new oncology combination therapies.
P31: Evidence Supporting Regulatory Withdrawals and Quantifying Their Impact on Public Health
10:45AM - 11:00AM
Lane S 1 , Lynn E2 , Slattery J3 , Shakir S2 1 Drug Safety Research Unit, SOUTHAMPTON, HAM, UK, 2 Drug Safety Research Unit, Southampton, Hampshire, UK, 3 European Medicines Agency, Amsterdam, ., Netherlands
OBJECTIVES: To assess trends in the use of evidence to support regulatory actions in Europe, and to identify a model which quantifies public health impact of a subset of these regulatory actions in terms of morbidity.
METHODS: Types of evidence used during regulatory decision-making were assessed for products withdrawn in Europe between 1999-2016 for safety reasons; patterns over time were evaluated. Quantification of impact of regulatory actions was performed for a subset of products (withdrawn/revoked in France/Germany/United Kingdom, 2012-2016). Annual exposures for each product were estimated using IQVIA Medical Research Data electronic health records. Reasons for regulatory action (adverse drug reactions [ADRs] of interest) were obtained from EMA records. Systematic review identified research quantifying ADRs, allowing estimation of absolute risk in exposed patients. Public health impact, expressed as annual number of ADRs prevented due to action, was estimated by modelling exposure and ADR risk.
RESULTS: In Europe between 1999 and 2016, 35 products were withdrawn; 91.4% of withdrawals (n=32) were supported by spontaneous reports and/or published case reports or case series, 60.0% (n=21) were supported by RCTs, 48.6% (n=17) were supported by non-randomized trials, 40.0% (n=14) were supported by observational studies. Meta-analyses contributed least frequently (n=11, 31.4%). The number of evidence sources cited in regulatory decisions increased over time, particularly for observational research. A sub-study involving 6 withdrawals/revocations for safety reasons in France, Germany and/or UK met inclusion criteria. Absolute risks were 0.1–41.25%. To estimate impact of withdrawal/revocation, average annual exposure was multiplied by absolute risk, providing crude numbers of ADRs prevented annually.
CONCLUSIONS: Spontaneous reports remain the most cited evidence source in regulatory decisions. However, a shift towards more epidemiological research supporting decision-making was observed. We successfully quantified public health impact of withdrawal/revocation in terms of serious morbidity. The method can be applied to products in other settings.
Spotlight Session
What Should Value Assessment of New Antibiotics Look Like in the Context of Pull Incentives? The How, What, and Where Next for Antimicrobial Resistance and Health Technology Assessment
Digital Conference Pass
Level: Intermediate
ISSUE: Antimicrobial resistance (AMR) continues to represent a substantial threat to global public health. New antimicrobials are desperately needed, but the market for new products is not commercially viable, largely due to stewardship principles keeping sales volumes low and the low cost of existing products. Appropriate value assessment and effective pull incentives are key to stimulating R&D in this area and bringing much needed new antibiotics to market.
Current methods of health technology assessment (HTA) focus almost entirely on health gains to a patient and costs to health care systems, thereby excluding the wealth of externalities offered by new antibiotics (such as reduced transmission and enablement of medical procedures, as well as further population level benefits). This exacerbates the already unappealing commercial environment for new antibiotics, discouraging the development of much needed new products.
One framework (STEDI) has been proposed and piloted in attempt to facilitate inclusion of externalities related to antibiotics within HTA. Yet, substantial confusion and apprehension remain around whether the framework should really be used for value assessment, and if so, how it can be operationalised.
Progress on the direction and methods for value assessment of antibiotics is urgently needed if the European Commission’s recent (April 2023) proposals on pull incentives for new antibiotics are to be effective.
OVERVIEW: Following a brief introduction to the issues by the moderator (4 mins), the panel will debate the future of value assessment of new antibiotics, including scope, methods and evidence, as well as its relevance in different countries and under different forms of pull incentive. Panellists will each speak for 12 minutes, providing their perspectives on the issues and articulating their arguments on the optimal direction of value assessment of antibiotics. 20 minutes will be reserved for audience discussion.
Moderators
Grace Hampson, MSc
Office of Health Economics, London, United Kingdom
Grace is an economist with expertise in the methods and processes of value assessment, reimbursement and healthcare decision making. Her current research interests include interactions between health technology appraisal and novel payment mechanisms, particularly in relation to innovative technologies such as cell and gene therapies and new drugs to tackle Antimicrobial Resistance. Grace leads OHE’s Policy Organisation and Incentives in Health Systems research team.
Panelists
Manuel Antonio Espinoza, MD MSc PhD
Pontificia Universidad Católica de Chile, Santiago, Chile
Manuel Espinoza is Associate Professor in the Department of Public Health and Head of the Health Technology Assessment Unit, both at Pontificia Universidad Católica de Chile. Manuel is also Editor-in-chief of Value in Health regional issues and Chair of the HTAi Latinamerica Policy Forum, and co-chair of the ISPOR SIG on Health Equity Research. He was president of the ISPOR Latinamerica Consortium, founder of the Chilean ISPOR Chapter, and member of the board of directors of ISPOR.
Manuel holds a medical doctor degree and Master in Epidemiology both from Pontificia Universidad Católica de Chile; a Master in Biostatistics from Universidad de Chile, and Master and PhD in Health Economics, both from University of York in the UK. Manuel´s work is focused on methods and processes for prioritization in health care as well as applied economic evaluation and impact policy analysis.
James Koh, PhD
National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
James Koh is a Scientific Adviser at the National Institute for Health and Care Excellence (NICE) and an Honorary Research Fellow at the Centre for Health Economics. His work covers novel science policy and research topics at NICE, including the evaluation of antimicrobials and complex diagnostics and its approach to economic perspective and inequalities in assessments.
James has previously worked in both consulting and academic roles, including as a Research Fellow at CHE between 2017 and 2021, specialising in the development of distributional cost-effectiveness analysis methods to incorporate inequality concerns into health economic analysis. He holds an MSc in Health Economics and PhD in Economics, both from the University of York.
Jorge Mestre-Ferrandiz, PhD
Independent Economics Consultant, Universidad Carlos III de Madrid, Madrid, Spain
Jorge Mestre-Ferrandiz is an independent health economics researcher and consultant since 2017, based in Madrid, Spain. He spent 15 years at The Office of Health Economics, starting as an Industrial Economist and finishing as Director of Consulting. Jorge has nearly 20 years’ experience consulting to the life sciences industry and related non-profit organisations and his clients and collaborators have included many global top 30 pharmaceutical companies, public sector organisations, international bodies, and a range of NGOs. He specialises in the economics of the industry, the pricing and reimbursement of medicines, the impact of public policy on R&D and innovation, R&D costs of new medicines, market access, the economics of rare diseases and orphan medicines, the economics of antimicrobial resistance (AMR), and pharmaceutical product portfolios. Recently he has co-authored two technical reports for the WHO Oslo Medicines Initiative.
Jorge started work on AMR and antibiotics back in 2007, where he participated at the two key conferences organised by the Swedish government in 2009 (during their EU Presidency), and in 2010. Since then, he has been involved at modelling push and pull incentives for new antibiotics, as well as the work that looked at the attributes of value for new antibiotics, which was then used by NICE for its pilot.
Jorge has a PhD in Economic Analysis from the University Autonoma of Barcelona, has published over 70 papers on health economics and is regularly invited to speak on related topics at academic and commercial international conferences. He is also an Associate Senior Research Fellow at the Science Policy Research Unit (SPRU) in the University of Sussex, a Visiting Fellow at OHE, member of the Editorial Board of Applied Health Economics and Health Policy, and member of the Advisory Board of Fundación Weber and Fundació HiTT respectively.
Case Studies
Breakouts: IP, WS, & OBS
Practical Issues in the Development of External Comparator Arms Using Real-World Evidence: Pragmatic Solutions or Unacceptable Uncertainties?
Digital Conference Pass
Level: Intermediate
PURPOSE: The development of external comparator arms based on real-world data (RWD) is often challenging due to inherent limitations in RWD resulting in substantial uncertainty. Alongside the extensive discussion of uncertainties (e.g., qualitative bias assessment), there is a need for consideration of pragmatic quantitative solutions to data limitations. In this workshop we discuss practical issues and potential pragmatic solutions to data limitations using two case studies and the effect of these on HTA decision making.
DESCRIPTION: Boglarka Szegvari will introduce the limitations of clinical trials for HTA due to trial design, changing treatment algorithms and geographical variation (9 minutes). While innovative trial designs might offer solutions, often RWD is required to fill the data gaps. RWD however is typically limited by the context, method, and purpose of the data collection. Noemi Muszbek and Neil Hawkins will present case studies where inherent data limitations required pragmatic solution (12 minutes each). Noemi Muszbek will discuss patient selection based on prognostic factors and predictors vs. statistical matching within an unanchored indirect comparison, the use of proxy variables for missing patient characteristics and the importance of clinical validations using a case study in cutaneous T-cell lymphoma. Neil Hawkins will talk about issues stemming from truncation and censoring in the survival data and the use of directed acyclic graphs (DAGs) for variable selection and evaluations using a case study in X-linked hypophosphatemia. David Epstein will talk about the opportunities and challenges posed by RWD for HTA (12 minutes). He will review current approaches used in Spain to assess clinical benefit and associated uncertainty, how RWD impacts pricing&reimbursement decisions, and how RWD might be considered in Joint Scientific Consultation.
The workshop will use real-time polling on the experience of audience members of the challenges/opportunities in using RWD and will be invited to share their experiences (15 minutes).
Discussion Leaders
Boglarka Szegvari, MSc
UCB Pharma, Brussels, WBR, Belgium
Bogi Szegvari, Msc, head of global payer strategy at UCB, responsible for the global value and access strategy for later-stage development assets. Her current area of focus includes equitable pricing, social business models and building multi-stakeholder collaborations around a common cause, including complex access & care pathways. She has over 20 years of experience in pharmaceutical market access, pricing, governmental affairs, health economics and outcomes research fields in various disease areas (oncology, neurology, hemophilia, gastroenterology, immunology) and technology types (biologics, vaccination & prevention programs, drug delivery device technologies).
Discussants
David Epstein, Phd
University of Granada, Granada, Spain
DAVID EPSTEIN is Professor at the Department of Applied Economics, University of Granada, Spain. His research centres on the economic evaluation of health care technology. He was special economic advisor to the Ministry of Health in Spain from 2021 - 2023 and participates as observer in the EU Joint Scientific Consultation subgroup
Neil Hawkins, MSc, PhD, MBA, CStat
University of Glasgow, Glasgow, United Kingdom
Neil Hawkins, MSc, MBA, PhD, CStat is a Professor of Health Technology Assessment at the University of Glasgow, Scotland, UK. Previously, he was Vice President leading the global Health Economics practice at ICON PLC and a member of the board of directors at Oxford Outcomes Ltd. He has post-graduate degrees in pharmacology, health economics and applied statistics. He has worked in the pharma, academic, and consultancy sectors in roles spanning basic pharmacological research, clinical development, epidemiology, and health technology assessment. His current research and consultancy focuses on: evidence synthesis and decision-analytic modeling; precision medicine, and estimation of subgroup effects; the use of health technology assessment analyses to inform technology investment and development; and the efficient allocation of capital in pharmaceutical development. He has taught ISPOR short courses since 2008, is a member of the Statistical Methods Special Interest Group, and was part of the Indirect Comparisons Task Force.
Noemi Muszbek, MSc
Visible Analytics, Oxford, Oxfordshire, United Kingdom
Noemi is Partner and Director of Health Economics at Visible Analytics, based in the UK. She has over 20 years of experience in consultancy, pharmaceutical industry and payer sectors. She has led the development of multiple economic models and programs of work, that had various components beside economic models, such as literature reviews, indirect comparisons, trial analyses, utility studies, retrospective database analyses and physician surveys; prepared and led submissions for technology assessment agencies across the world; written white papers in different policy issues and has taken part in guideline development for economic evaluation and for reimbursement process.
Member Group Meetings
Digital Health Special Interest Group Open Meeting
Are you interested in the interaction between health outcomes research and Digital Health? Come meet the Digital Health Special Interest Group leadership team on Tuesday, November 14
th at 10:15AM and be the first to hear about our stimulating key project findings and help guide our future efforts.
Specifically, we will cover the PICOTS-ComTeC Framework for defining digital health interventions including the SIG activities at this conference – a research podium session , a workshop , and propose an exciting new key project for 2024 to develop a CHEERS extension for digital health. You can also learn about our concluding webinar on data privacy coming up in December and topics for future webinars/journal clubs. We will announce the timeline for the opening of our Digital Health SIG online community where you can find resources and be part of the conversation on Digital Health topics.
Looking forward to seeing you there!
Breakouts: IP, WS, & OBS
The Elephant in the Room: Which Stakeholders Should be Responsible for RWE Generation After Launch?
Digital Conference Pass
Level: Intermediate
ISSUE: There has been a sustained growth of real-world data (RWD) sources for research purposes that has contributed to the increased openness to RWE studies in regulatory and HTA decision-making; however, the logistical and financial costs needed to generate high-quality RWE from these sources hampers the availability of RWE for decision-making. Although the responsibility in certain situations (such as in post-authorization safety studies) clearly falls on the manufacturers, it is currently not clear who should be responsible in other scenarios (e.g., filling evidence gaps needed by payers post-launch). This session will provide a forum to debate, from a variety of different perspectives, whose responsibility it is to generate RWE to address evidence gaps, and to discuss potential mechanisms to foster constructive dialogue between stakeholders when determining this responsibility.
OVERVIEW: The moderator will provide an overview of the stakeholders, different scenarios of when ownership of RWE generation is unclear, and challenges associated with RWE generation (10 minutes). The diverse panelists will then present their perspective on when key stakeholders should and/or should not be responsible for RWE generation (10 minutes each). The panels will also discuss ideas and incentives for collaboration among stakeholders to address evidence gaps. The moderator will then oversee a debate between the panelists and incorporate questions/comments from the audience (20 minutes). Stakeholders (regulators, researchers, industry, HTA bodies/payers, patient advocates, etc) interested in using RWE for decision-making will benefit from attending.
Moderators
Ashley Jaksa, MPH
Aetion, Inc, Boston, MA, USA
Ashley Jaksa is Market Access Scientific Strategy Lead at Aetion. She focuses on collaborating with decision-makers to enhance their use of RWE and setting methodological guidance for RWE generation. She also works with Aetion’s biopharma clients on incorporating RWE in their global market access strategy. She has presented her research at numerous international conferences including ISPOR, HTAi, and AMCP, and published her findings in various peer-reviewed publications. She holds a bachelor's degree from the University of Michigan-Ann Arbor and a MPH from Yale University.
Panelists
Solange Corriol-Rohou, MD
Pharmaceutical Company: AstraZeneca, GMD, Paris, 75, France
Solange Corriol-Rohou, a pulmonologist/immuno-allergist by training, joined AstraZeneca R&D in 2004 and is currently Sr. Regulatory Affairs & Global Policy Director, with responsibilities in the Respiratory/Infection and Vaccine/Immune franchises. Over the past 20 years, moving from the French Medicines Agency/EMA and academia to the pharmaceutical industry, she has gained strategic experience in drug development. She is quite active within EFPIA, ICH and IMI/IHI, and passionate about paediatric drug development.
Pall Jonsson, BSc, PhD
Data and Analytics, National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
Pall Jonsson is Programme Director for Data and Real World Evidence at the National Institute for Health and Care Excellence (NICE). His team has a strategic role in ensuring NICE is at the forefront of harnessing new and emerging opportunities for using real world data to inform NICE’s guidance to the health and care sectors.
Pall has a PhD in bioinformatics from the University College London. Before taking up his current role, he was Associate Director for Science Policy and Research, responsible for NICE’s portfolio of international research projects in areas such as big data and real-world evidence. Earlier experience includes work in academia, biotech and the pharmaceutical industry.
Jesper Kjær, MSc
Danish Medicines Agency, Copenhagen, Denmark
Director of The Data Analytics Centre at the Danish Medicines Agency and co-chair of HMA / EMA Big Data Steering Group
Issue Panel on the Identification and Implementation of Clinically Meaningful Thresholds for Clinical Outcome Assessments: Challenges and Considerations
Digital Conference Pass
Level: Intermediate
ISSUE:
Clinically meaningful change remains a topic marked by debate about how we can best define, determine and deploy this concept with clinical outcome assessments (COAs). This session will address key challenges and considerations, including both the scope for harmonization and issues relevant to emerging areas such as digital endpoints.
OVERVIEW:
It is now widely recognized that treatment benefits should correspond to changes/outcomes that are tangible and meaningful to patients. It is consequently becoming more common for key trial endpoints to be defined with reference to clinically meaningful thresholds (CMTs) on COAs. The conceptualization, identification and implementation of CMTs is an area that remains marked by diversity of perspective and approach, resulting in on-going debate and development. These developments are most recently reflected by changes in terminology and approach flagged in FDA’s 2023 draft guidance on ‘Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making’, which stands in contrast to other frameworks/strategies such as IQWiG’s universal 15% threshold. Concurrently, the emergence of digital health technologies as novel COA modalities poses new questions and hurdles. We are bringing together thought leaders in this field (representing different stakeholders) to discuss key issues on this topic. The session will commence with an overview [5 min] of the primary concepts (conceptual and methodological) and general sources of heterogeneity/variability in CMTs. Two invited speakers will then present on:
The identification and implementation of CMTs within oncology, and the importance of harmonization efforts (such as that led by SISAQOL-IMI) [15min] The novel insights and new challenges in the assessment of meaningful change with digital endpoints [15min]. This will be followed by a discussant [10 min] and Q&A [15 min]. Through the different perspectives offered, this panel will serve to convey (and aspire to inform) the evolving understanding/practice in this important area of clinical outcome research.
Moderators
Dara O'Neill, PhD
IQVIA, Barcelona, N/A, Spain
Dara is a health researcher and psychometrician with over 15 years’ experience in both academic and professional settings. As part of the Statistics and Psychometrics’ team at IQVIA’s Patient Centred Solutions, she has led diverse projects on the evaluation and modification of clinical outcome assessments. This work has been in particular focussed on patient reported outcome measures and efforts to establish clinical meaningfulness of reported change in interventional settings. Prior to joining IQVIA, Dara was a Senior Research Fellow at University College London, overseeing two separate programs of cross-study data harmonization, including one in support of the UK National Core Studies’ response to COVID-19.
Panelists
Johannes Giesinger, PhD
Medical University of Innsbruck, Innsbruck, Austria
Dr Johannes Giesinger is an assistant professor at the Medical University of Innsbruck, in Austria, specialising in patient-reported outcome (PRO) research. Dr Giesinger has been leading a number of international projects focusing on methodological aspects of the development and use of PRO measures. Currently, he engages in studies facilitating the interpretation of PRO data collected in clinical trials and daily practice. This includes, for example, his work within the SISAQOL-IMI consortium that is developing guidelines for the statistical analysis of PRO endpoints in clinical trials.
Laura Lee Johnson, PhD
Food and Drug Administration, Silver Spring, MD, USA
Dr. Laura Lee Johnson is a division director in the Office of Biostatistics at the U.S. Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER) and the Office of Translational Sciences’ Patient Focused Drug Development liaison. Among her many activities Dr. Johnson serves on the FDA-NIH Interagency Clinical Outcome Assessments Working Group, the IMI PREFER Scientific Advisory Board, and co-directs the NIH Principles and Practice of Clinical Research course.
Marie Mc Carthy, BSc, MSc, MBA
Novartis, Dublin, D, Ireland
Marie has worked within the digital endpoint sphere for more than a decade, supporting the optimal use of these endpoints in research and drug development. She has spent these years evangelizing the value and benefit of digital endpoints derived from DHTs and ensuring operational excellence in implementing these technologies by developing robust operational frameworks. Her current role at Novartis is focused on supporting the appropriate selection and validation of digital endpoints in drug development programs. Before joining Novartis, Marie was part of the Innovation team at ICON PLC, focused on selecting, identifying, and using novel and innovative DHTs and derived digital endpoints.
The Place of Artificial Intelligence in HTA and HEOR
Digital Conference Pass
Level: Intermediate
ISSUE:
Unlike other industries, use of AI/ML in HTA is widely considered to be in its infancy HTA bodies are beginning to recognize the value of AI/ML, such as in the selection of covariates in survival models, but use cases are few Existing concerns around the use of AI/ML in HTA include questions on appropriateness, transparency, and accessibility to non-experts However, AI/ML methods have proven advantages over traditional approaches in cases of multi-variable datasets often seen in healthcare How then can the benefits of these methodologies be integrated with concerns of decision makers for a balanced approach in HTA and HEOR? OVERVIEW:
(5 min) Moderator Ines Guerra will introduce the topic of AI/ML in HTA, explain the current situation and important background. She will outline the advantages and disadvantages of AI/ML methods and key vocabulary for the audience.
(10 min) Pearl Gumbs (Industry) will build on this to explain how AI/ML may benefit manufacturers in value demonstration, noting that clear guidance on acceptability of AI/ML is required to reduce rejection risk.
(10 min) In response, Mariam Sood (Payer) will acknowledge the positives of AI/ML in improving process timeliness but explain concerns for AI/ML in HTA. She will detail what needs to change for better applicability in HTA and conclude with a future perspective on use of AI/ML methods from the view of NICE. (10 min) Finally, Noemi (Academic) will explain how typical challenges of AI/ML (like lack of transparency) can be overcome, and the role of academia in this advancement. She will explain how academic innovation will help bridge Industry and decision makers to embed AI/ML in HTA.
(25 min) Facilitated audience discussion, with opportunities to ask questions and propose solutions to accelerate use of AI/ML in HTA Stakeholders: AI/ML developers, Payers, Industry HEOR/RWE, Market Access professionals
Moderators
Ines Guerra, MSc
IQVIA, London, United Kingdom
Ines is a Senior Principal at IQVIA leading numerous projects in HEOR (cost-effectiveness and budget impact models, trial data analysis, indirect treatment comparisons, global value dossiers, systematic literature reviews, surrogacy analysis and HTA submissions). Ines is currently involved in several projects exploring AI/ML in the context of HEOR and HTA. Ines holds an MSc Honours in Computers Engineering from the New University of Lisbon and an MSc in Health Economics, Policy and Management from the London School of Economics and Political Science.
Panelists
Lorna Dunning, MBiochem MPH
NICE, London, LON, United Kingdom
As a Senior Technical Adviser - Methods, Lorna provides technical and methodological leadership to the TA/HST programme at NICE. Lorna has led a number of transformational projects including the development and implementation of the CHTE 2022 methods update into health technology evaluation practice.
Lorna holds a Masters degree in Biochemistry from University of Oxford, and a Masters in Public Health from the University of Cape Town (South Africa). Before joining NICE, Lorna worked as a researcher at the Medical Practice Evaluation Center (Massachusetts General Hospital, USA) leading a WHO project to use model-based approaches to inform guidance on paediatric HIV at international and country levels.
Pearl Deborah Gumbs, PhD
Boehringer Ingelheim, Ingelheim am Rhein, Mainz-Bingen, Germany
Experienced Senior Manager with a demonstrated history of working in the pharmaceuticals industry. Skilled in Epidemiology, Vaccines, Oncology, Databases, and Health Technology Assessment (HTA). Strong professional with a PhD focused in Pharmacoeconomics and pharmaco-epidemiology from Universiteit Utrecht.
Noemi Kreif, PhD
Centre for Health Economics, University of York, York, North Yorksire, United Kingdom
Dr Kreif’s research is centred around incorporating methodological innovations from statistics, causal inference and machine learning into health economics and health policy research. Her most recent methodological research project developed a causal machine learning framework to estimate treatment effect heterogeneity of health policies, and linked these estimates with decision models to provide optimal treatment recommendations. Dr Kreif was part of the ISPOR task force which authored the “PALISADE Checklist” and Good Practice Report on Machine Learning Methods in Health Economics and Outcomes Research. Prior to joining the Centre for Health Economics, Dr Kreif was a lecturer at the London School of Hygiene and Tropical Medicine, where she previously received her PhD (2013). Previously, she worked at an international pharmacoeconomics consulting company. Dr Kreif has published widely in leading health economics and statistics journals, including Health Economics, Journal of Health Economics, American Journal of Epidemiology and Biometrics.
Wouldn't It Be NICE? Could Europe Accept the Consequences of the US Adopting the UK's CEA Framework for Capping Launch Prices?
Digital Conference Pass
Level: Intermediate
ISSUE: This panel will explore the potential consequences for Europeans were the US to adopt NICE’s CEA framework for reducing its willingness to pay for novel medicines and whether they might prefer NICE modify those methods before the US imports them.
OVERVIEW: Policymakers in the US are increasingly willing to rely on CEA to determine the value of new medicines and make reimbursement decisions. They are eager to understand why European countries enjoy a better “deal” on drug prices. There is a parallel debate about whether the CEA framework in the UK is too narrow, excluding many elements some argue should be included in a fuller, generalized CEA calculation. European payers are more willing than their US counterparts to restrict access to treatments based solely on cost, but Europeans may not truly appreciate the consequences of their lower willingness to pay. That’s because, today, the US market pays more for new medicines, supporting positive expected returns on R&D investment, so Europeans still can count on access to new medicines eventually (e.g., at worst, after they go generic), regardless of how they value them. But industry warns that they can’t justify investing in R&D if they have to assume that products that emerge from their R&D portfolios would be capped by the US at prices set by NICE’s math. Discussion will include: A) whether worthwhile innovation would be impacted by adoption of NICE’s CEA by the US; B) if so, would the UK and rest of Europe accept those consequences or prefer to see NICE’s math revised; C) if revised, how.
NOTE: There will be a discussion group on this topic following the session. Join us in the Discussion Lounge in Hall E North from 11:45-12:45 in Discussion Group B.
Moderators
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of stratified medicine for healthcare payers and developers; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A Visiting Professor in Practice at the London School of Economics and a Visting Fellow at the Center for Global Development, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
Panelists
Peter Kolchinsky, PhD
RA Capital, Boston, MA, USA
Peter Kolchinsky is Founder and Managing Partner at RA Capital, investing in companies developing drugs, devices, and diagnostics. He serves as a board member for various public and private companies. Peter served on the Board of Global Science and Technology for the National Academy of Sciences and is the author of “The Great American Drug Deal” and “The Entrepreneur’s Guide to a Biotech Startup.” He founded the nonprofit No Patient Left Behind to advocate for healthcare reform and promote innovation. Peter holds a BA from Cornell University and a PhD in Virology from Harvard University.
Steven Pearson, MSc, MD
Institute for Clinical and Economic Review, Boston, MA, USA
Steven D. Pearson, MD, MSc is the Founder and Special Advisor of the Institute for Clinical and Economic Review (ICER), an independent non-profit organization that evaluates the evidence on the value of medical tests, treatments, and delivery system innovations to encourage collaborative efforts to improve patient care and control costs. Dr. Pearson is also a Lecturer in the Department of Population Medicine at Harvard Medical School. He received his medical degree from UCSF, completed an internal medicine residency and research fellowship at Brigham and Women’s Hospital, and obtained a Master of Science Degree in Health Policy and Management at the Harvard School of Public Health.
Beth Woods, BA, MSc
University of York, York, United Kingdom
Beth is a Senior Research Fellow in the Team for Economic Evaluation and Health Technology Assessment (TEEHTA) and has been based at the University of York since 2014. Beth holds a BA in Economics from the University of Cambridge and an MSc in Economic Evaluation in Healthcare from City University.
Prior to joining CHE Beth was a Director in the Health Economics team at Oxford Outcomes, a private consulting firm, where she specialised in applied economic evaluation from 2006-2013.
Beth has worked on economic evaluations of a range of technologies, including the application of advanced statistical and decision modelling methods. Beth has also contributed to methods in the field, in particular relating to model structuring in oncology, evaluation of pharmaceutical pricing policy, and evaluation and pricing of technologies to address antimicrobial resistance.
Beth is a member of the NIHR HTA Clinical Evaluation and Trials Board and sits on the Board of Directors for the York Health Economics Consortium.
Uncertainty Management in Drug Evaluations of ATMPs: From Evidence to Reimbursement & Payment Models
Digital Conference Pass
Level: Introductory
ISSUE: To allow for early authorisation and aim for early patient access, marketing authorisation applications for ATMPs are often granted whilst the evidence is still immature. Assessing these medicines has therefore posed challenges for regulators, Health Technology Assessment (HTA) decision-makers and healthcare payers. To collect additional information and mitigate the financial risk of reimbursing these therapies under large uncertainties, outcome-based reimbursement and delayed-payment models are frequently debated as promising strategies to create win-win-win situations form a patient, industry and healthcare payer perspective.
OVERVIEW: The main underlying uncertainties in the evidence supporting ATPMS from a regulatory and HTA perspective will be presented (15 min) and how the type of uncertainty and level of expected added benefit can be used to determine which kind of managed entry agreement is most suitable (15 min). The panellist with an patient, industry, healthcare payer and HTA perspective (30 min) will discuss how early dialogues could support mutual understanding of the most pressing underlying uncertainties and how horizon scanning can play a valuable part in informing and preparing stakeholders throughout the life cycle of ATMPs. Moreover, the discussion will go into what role the new EU HTA regulation could take in supporting uncertainty management and payment and reimbursement decision-making and provide perspective on implementing outcome-based reimbursement models in the future .
Audience participation will include discussion on how clinical uncertainties early in the life cycle of ATMPs have a rippling down effect to later stages in the payment and reimbursement decision-making, what guidance is needed to ease reimbursement decision-making and how the current and future policy landscapes support the ability to create win-win-win situations for patients, manufactures and healthcare payers for the upcoming ATMPs. This interactive and informative panel will be valuable to regulators, HTA agencies, healthcare payers, policymakers, researchers and industry analysts interested in payment and reimbursement strategies. .
Moderators
Wim Goettsch, PhD
National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He has serves as a special HTA advisor to Zorginstituut Nederland and a former director of EUnetHTA JA3 Directorate.
Panelists
Marcelien Callenbach, MSc.
Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht, UT, Netherlands
Marcelien Callenbach is a PhD candidate at Utrecht University in the Division of Pharmacoepidemiology and Clinical Pharmacology. Her research focuses on managed entry agreements for innovative therapies with large clinical- and/or cost-effectiveness uncertainties, in which she combines her background in Health Economics, Policy and Law, MSc. and Political Sciences, BSc.
Francois Houyez, Patient Advocate
Rare Diseases Europe, Paris, France
François Houÿez is Director of Treatment Information and Access at the European Organisation for Rare Diseases EURORDIS.
He has always been working as a patient advocate since the early 90s, first in the HIV/AIDS advocacy, and in rare diseases since 2003.
His experience with compassionate use programmes started in 1988.
He pioneered patient advocacy with the European Medicines Agency as part of the first patients’ delegation that engaged dialogue with the Agency back in 1996.
François is also a patient.
Mohit Jain, PhD, MBA
BioMarin Pharmaceutical Inc, London, SRY, United Kingdom
Mohit is responsible for Global Value, Access & Strategic Pricing in BioMarin. BioMarin is a company focused on developing life transforming therapies for patients suffering genetic diseases with a number of gene therapies in development. Mohit has been with BioMarin for over twelve years and prior to this worked in other companies including large pharma and consulting. By background a scientist and then worked in corporate finance before finding a calling in Market Access. Mohit is married with two lovely girls aged 14 and 16 years, occasionally trying to fit in a run.
Marc van de Caastele, PhD
RIZIV-INAMI, Saint-Josse-ten-Noode, Brussels Region, Belgium
Case Studies
HTA Case Studies I
These case studies involve the evaluation and implementation of health technologies, particularly pharmaceuticals, in various healthcare systems. They highlight the challenges and strategies related to decision-making, cost containment, access, and evidence-based evaluation for new treatments in different countries.
Moderator
Anne Sørensen, PhD
The Danish Health Technology Council, Aalborg, North Denmark, Denmark
Health economist at the Danish Health Technology Council, PhD in health economics and a masters degree in medical market access.
CS11: Utilizing Health Technology Assessment (HTA) to Develop Health Technology Management (HTM) Processes Under a Life-Cycle Approach to Drug Reimbursement in Ireland
10:30AM - 10:45AM
Gorry C 1 , Doran S1 , Duggan B 2 , Smith A3 , Clarke S1 , Barry M1 1 HSE Medicines Management Programme, Dublin, Dublin, Ireland, 2 HSE Medicines Management Programme, Dublin 7, Ireland, 3 HSE Medicines Management Programme, Dublin 8, Ireland
Problem Statement: In Ireland, new medicines are increasingly reimbursed for sub-populations of the licensed indication, due to high-cost, budgetary constraints and low quality clinical evidence. Health Technology Assessment (HTA) can identify sub-populations where reimbursement may bring greatest benefits; health technology management (HTM) protocols are developed around these recommendations to ensure utilisation is limited to the approved population.
Description: The Health Services Executive (HSE) in Ireland manages medicines expenditure within the publically funded health system. A variety of HTM processes have been developed to implement managed access to medicines including the following:
automated validations implemented via the national reimbursement claims system;
reimbursement application systems;
managed access protocols; and
managed access protocols including outcome data collection.
Choice of process is determined by clinical aspects of the treatment and target population, setting and route of administration, feasibility, existing controls in the therapeutic area, commercial terms etc., which are largely informed by the HTA report. The final process is determined in conjunction with some or all of the (i) HSE-Corporate Pharmaceutical Unit, (ii) HSE-Primary Care Reimbursement Service, (iii) HSE-Acute Hospitals Drug Management Programme, (iv) relevant HSE-National Clinical Programme.
The HSE-Medicines Management Programme (HSE-MMP) will present case studies of each approach, demonstrating (i) how HTA reports inform HTM approaches, (ii) which approaches are most suitable for particular drug types, (iii) IT infrastructure supporting the approach, and (iv) lessons learned in implementation to date. Case studies include medicines for atopic dermatitis, migraine, obesity, cardiovascular disease and dermatology and rheumatology indications.
Lessons Learned: HTM provides an effective mechanism to facilitate access to high-cost medicines for targeted patient groups, while providing increased oversight and budgetary certainty. Cost certainty provided by HTM permits avoidance of HTA in certain therapeutic areas. Early consideration of the HTM approach during the HTA process supports subsequent successful implementation post-reimbursement.
Stakeholder perspective: HSE-Payer/Government Organisation
CS12: Data Characteristics and Uncertainties in Assessments of New Medicines at the Danish Medicines Council
10:45AM - 11:00AM
Dehlendorff C1 , Johnsen HM2 , Gudmann N 3 , Jensen JD2 , Galijatovic E4 1 Danish Medicines Council, Copenhagen, Copen, Denmark, 2 The Danish Medicines Council, Copenhagen, Copenhagen, Denmark, 3 Danish Medicines Council, Copenhagen, Denmark, 4 Danish Medicines Council (DMC), Solrød Strand, Denmark
Problem Statement: Effect sizes and level of certainty are important factors for decision making regarding implementation of new treatments. For medicines, health technology assessments can be challenged by limitations in the available evidence. The purpose of this study was to characterize the type of data available in dossiers for medicines assessed by the Danish Medicines Council (DMC).
Description:
Lessons Learned:
54 % of studies providing a hazard ratio for overall survival showed a statistically significant effect on overall survival. Hazard ratios were primarily available in dossiers for cancer treatments.
The DMC’s health economic assessments were based on cost utility analysis (61%), cost minimization (33%) or other types of economic evaluation (6%). The likelihood of a positive recommendation tended to be higher in assessments based on phase III studies with a direct comparison (52% vs. 35%).
Indirect comparisons and early phase studies are associated with a greater risk of bias and uncertainty regarding efficacy and safety. Our analysis shows that the DMC often have to make decisions about implementation of new medicines based on limited evidence, which may lead to suboptimal decisions.
Stakeholder perspective:
CS10: HTA Reforms in Germany: Implications for Pharmaceutical Pricing and Reimbursement in Germany and the EU
10:15AM - 10:30AM
Matthijsse S 1 , Akehurst R2 , Gladwell D2 , Wasem J3 1 Lumanity, Sheffield, DBY, UK, 2 Lumanity, Sheffield, South Yorkshire, UK, 3 University of Duisburg-Essen, Essen, Germany
Problem Statement: Germany approved a cost-containment bill in the final quarter of 2022. We explored the most significant implications for pharmaceutical pricing and reimbursement in Germany following the approval of the cost-containment law and how the new bill interacts with the new European Regulation on HTA.
Description: We interviewed the former chairperson of the Arzneimittelmarkt-Neuordnungsgesetz (AMNOG) Drug Price Arbitration Board Juergen Wasem, and reviewed how the new cost-containment law changes the way price negotiations were traditionally done in Germany. By combining these changes with the process of the new European Regulation, we came to a consensus view on the likely interaction between these new developments within the EU landscape.
Lessons Learned: In Germany, the most significant changes affect the guidelines on free pricing period, free set list price, pricing rules per level of benefit, price-volume agreements, pricing for combination products, wastage, and orphan drug appraisal. The three main factors that are considered for pricing are European prices, the price of comparable compounds in Germany, and the believed monetary value of the additional benefit. We believe that the extent to which Germany will comply with the pan-European regulation will largely depend on whether the EU Regulation implementation working groups sufficiently acknowledge the new German practices. As a result, we may either see a future appraisal landscape in Europe much like that in Germany, or a persistent divergence in the way Member States implement the Regulation. The German approach to evaluating drugs may influence the countries with lower healthcare budgets less, as the question of reimbursement and access remains in the hands of the individual Member States. For these Member States, the results of the Pharmaceutical Strategy of the European Commission may be more relevant.
Stakeholder perspective: Industry, government-affiliated stakeholder
10:30 - 13:30
Poster Session 3
Presenters will be with their posters from 12:30 - 13:30
11:15 - 13:15
Lunch
11:30 - 12:00
Fast Facts
Pathways to ISPOR Leadership
Become an ISPOR Leader! ISPOR members help the Society realize its mission of improving healthcare decision-making globally through their volunteer participation in member groups, councils, task forces, strategic initiatives, and special interest groups. The volunteer leaders who participate in ISPOR groups benefit greatly from the enhanced exposure and personal development that their participation affords them and ISPOR gains the knowledge and expertise that our expert members provide. During this session, gain quick insight into the pathways to lSPOR leadership through shared stories, experiences, and discussion.
Discussion Leader
Lotte Steuten, PhD
Office of Health Economics, London, Greater London county, United Kingdom
Lotte Steuten, PhD, is Deputy Chief Executive of the not-for-profit Office of Health Economics (OHE) and the scientific and business lead for its international research-led work program—maintaining its reputation for objective, innovative, and high-quality research, and meeting its charitable goals. She is also an Honorary Visiting Professor at City, University of London (UK) contributing to its teaching programmes in health economics.
For more than 20 years, Lotte has worked in academic, research, and consulting HEOR roles with one focus — improving healthcare decision-making through high-quality research and analysis. To achieve that, she has collaborated with HTA authorities, pharmaceutical and medical device companies, patients, payers, policymakers, academic researchers, clinical specialists, and venture capitalists. She has gained broad HEOR expertise of various technologies: precision medicine, oncology, vaccines, antibiotics, diagnostics, digital health, and medical devices.
Lotte’s career has been based in the United States, the Netherlands, and the UK. Collaborations with HEOR experts in Southeast Asia, Africa, and Latin America have provided her with deep insights into the differences and commonalities between the role of HEOR in different healthcare systems, cultures, and societies. With this background, she has: led diverse international teams; developed strategies and delivered on program and organisational missions, values, and objectives; acquired research funding and managed budgets; and been responsible for executive decision-making as well as legal and fiduciary matters.
As an active ISPOR member since 2004, Lotte has served on various Taskforces and Committees, delivered Short Courses, was co-Chair of ISPOR EU 2021, and is currently a member of the Board of Directors, and an Associate Editor for Value in Health. She aims to be a role model for diversity and played an active role in the 'Women in HEOR' initiative.
Before joining OHE, Lotte worked in Seattle (US) as an Associate Professor at the University of Washington and at the Fred Hutch Cancer Research Center. She graduated cum laude with her PhD from Maastricht University (NL) and then worked at Brunel University (UK) and Twente University (NL).
11:30 - 12:15
Poster Tours
EU Joint Clinical Assessment Poster Tour
Posters featured in this tour:
PT25: Comparison of Approval Dates of New Substances between the United States of America (FOOD AND DRUG ADMINISTRATION, FDA) and Europe (EUROPEAN MEDICINES AGENCY, EMA)
PT26: How Aware Are Biotech and Pharmaceutical Companies of the Implementation of the New EU HTA?
PT27: How Much Does TLV Value Rarity? A Review of Reimbursement Decisions on Orphan Drugs in Sweden from 2017 through 2022
PT28: Lost in PICO? a Simulation of the EU HTA Scoping Process
PT29: Predicting PICOs for EU Joint Clinical Assessment: Lessons from PICOs in Relative Effectiveness Assessments (REA) from EUnetHTA Joint Action 3 Project
PT30: Will Health Technology Assessment (HTA) Bodies in Europe Accept Evidence from an External Control Arm to Supplement Evidence from Clinical Trials for Chronic Diseases?
Sponsor
Moderator
Melinda Goodall, MSc PhD
Goodall HTA Consulting, Manchester, Manchester, United Kingdom
Oncology Poster Tour
Posters featured in this tour:
PT31: A Time Series Analysis of Immune Checkpoint Inhibitors Use in Italian Population: 2017-2022
PT32: Cost-Effectiveness of Lorlatinib in Second-Line Treatment of ALK-Positive Advanced Non-Small Cell Lung Cancer in China
PT33: Cost-Effectiveness of Pembrolizumab Plus Chemotherapy, With or Without Bevacizumab for the First-Line Treatment of PD-L1-Positive Patients With Persistent, Recurrent, or Metastatic Cervical Cancer in France
PT34: Impact in Health Outcomes of PD-(L)1 Inhibitors to Treat Early Stages Cancers in Switzerland
PT35: Matching-Adjusted Indirect Comparison (MAIC) of Dabrafenib Plus Trametinib Versus Pembrolizumab Plus Chemotherapy in Patients with Treatment-Naive Metastatic BRAF V600 Mutation-Positive Non-Small Cell Lung Cancer (NSCLC)
PT36: Real-World Analysis on Therapeutic Pattern and Economic Burden of Androgen-Receptor Signaling Inhibitors (ARSI) and Taxane-Based Chemotherapy Treated Metastatic Castration-Resistant Prostate Cancer Patients in Italy
Sponsor
Moderator
Rose Wickstead, MPharm MSc (Health Economics), PGCert (Clinical Pharmacy)
Costello Medical, London, United Kingdom
11:45 - 12:15
Exhibit Hall Theater
Are You Scoping Ready for the New EU HTA Regulation Implementation?
Digital Conference Pass
The European Union’s new regulation for health technology evaluation (Regulation (EU) 2021/2282), focusing on reducing duplication of efforts by national HTA bodies and ensuring efficient use of resources, will be applied starting in January 2025 with oncology treatments and advanced therapy medicinal products (ATMPs), with full implementation in all therapeutic areas by 2030. This regulation affects all companies wanting to market products in the EU, so it is critical to be adequately prepared for new submissions.
One significant consideration, and potential challenge, to anticipate is Population, Intervention, Comparator, and Outcomes (PICO) scoping. Given the short timeframe between scoping and submission, companies developing healthcare products will need to identify the most likely PICOs and do the associated analytics well in advance. This session will discuss some of the challenges, issues and considerations when preparing for this major step of the new EU HTA process, including:
The number of potential PICOs Variable levels of management within companies Key tradeoff decisions in trial design and operations
Additionally, the speakers will compare the new EU HTA approach with NICE’s approach for perspective and will discuss ways to optimize success with the new submission process moving forward. Please join us to better understand how to effectively prepare for your HTA submissions moving forward!
Sponsored by Evidera, part of PPD
Sponsor
Evidera, part of PPD
Speaker
Jessica Griffiths, BSc, MEnt
Evidera, part of PPD, London, United Kingdom
Jessica Griffiths, MEnt, BSc, is a Senior Consultant within the Evidera Value and Access team in London. Jessica has over nine years’ consulting experience with experience in a wide range of projects including evidence planning, launch sequence planning, funding pathway mapping, competitive landscape analysis, HTA tracking, launch pricing, value message testing, Integrated Scientific Advice strategy and payer advisory boards/workshops.
Jessica has significant experience with pricing and market access in the EU and US, in addition to experience in all major markets including LATAM, Asia, Canada and Australia. She has a broad range of project experience in cardiology, haematology, oncology, immunology, nephrology, rheumatology, neurology, pain management and rare diseases. She is the UK expert and co-lead of the Evidera policy trends initiative and develops up-to-date reports on pricing and market access policy developments across the EU, UK, US, Canada, Australia and Japan.
Prior to joining Evidera, Ms. Griffiths worked as an analyst at IQVIA, with a particular focus in pricing and market access. She worked with many of the top 10 Pharma companies, conducting research in global markets and multiple disease areas.
Prior to IQVIA, Ms. Griffiths worked as a business development analyst at the Cell Therapy Catapult. She holds both a chemistry bachelor's and chemistry and enterprise master's from Manchester University.
Paulina Rolska-Wójcik, PhD
Evidera, part of PPD, Warsaw, Poland
Paulina Rolska-Wójcik is a Director within Evidera’s Value and Access Consulting team bringing over 11 years of experience in healthcare, including both industry and consulting roles. In current role at Evidera, she is actively involved in various types of projects, including Scientific Advice, HTA landscape assessments, and projects related to EU HTA regulations for global and local pharma teams. Paulina Rolska-Wójcik has led projects with a focus on launch sequence strategy projects, product value testing and market access opportunities assessment including many markets (e.g., EU4+UK, US, Poland, Czech Republic, Hungary, Slovakia, Slovenia, Croatia, Albania, Greece, Romania).
11:45 - 12:45
ISPOR Forums
ICHOM and ISPOR's Efforts to Further Patient-Centered Outcomes Research
Founded in 2012, the International Consortium for Health Outcomes Measurement (ICHOM) has made significant impact on defining outcomes measurement sets for major diseases, with the goals of informed decision making, quality improvement, and reduced costs. ISPOR shares these goals and has also had major impact on outcomes research methods, particularly in the context of value assessment. This session will describe the complementary activities of both organizations and explore how together we can further the use of scientifically rigorous outcome measures in health care decision making.
Moderators
Brian O'Rourke, PharmD
ISPOR HTA Council, Ottawa, ON, Canada
Dr. Brian O’Rourke is the President of ISPOR for the period from July 2023 to June 2024. He served as the President and Chief Executive Officer of CADTH from 2009-2020. He joined CADTH following a distinguished career as a Pharmacist and Health Care Executive with the Canadian military. With over 40 years of experience in health care, Brian is a leading expert in the science and practice of health technology assessment (HTA) and served as the Board Chair for the International Network of Agencies for Health Technology Assessment from 2014 to 2018. He has a Bachelor of Science in Pharmacy from Dalhousie University and a Doctor of Pharmacy from the University of Toronto. Dr. O’Rourke continues to play an active role in the global HTA community. He is Chair of the Health Technology Assessment Steering Committee and a member of the Scientific Advisory Council at the Centre for Innovation in Regulatory Science (CIIRS). In November 2020, Dr. O’Rourke was appointed Colonel Commandant (Honorary) of the Royal Canadian Medical Service.
Speakers
Jennifer Bright, MPA
ICHOM, Alexandria, VA, USA
Jennifer Bright, MPA (she/her) is President, International Consortium for Health Outcomes Measurement (ICHOM), a nonprofit that works with patients and clinical experts worldwide to define and deploy standardized tools to measure outcomes that matter to patients. Prior, she held executive roles at the Innovation and Value Initiative (IVI), focusing on advancing patient-centricity, transparency and equity in value assessment.
Jennifer is President, Momentum Health Strategies®; Board Chair, Mental Health America; and editor, American Journal of Accountable Care. She holds degrees in political science and public administration from Trinity University and George Washington University.
Jens Grueger, PhD
Boston Consulting Group, Zurich, Switzerland
Jens Grueger is a Director and Partner at the Boston Consulting Group based in Zurich Switzerland and a Fellow of the BCG Henderson Institute. He provides expert advice to companies in the life science industries on pricing, access and affordability. Since July 2019, he is an Affiliate Professor of Health Economics at the CHOICE Institute at the University of Washington School of Pharmacy in Seattle, USA. He was the 2020-2021 ISPOR President, the leading professional society for health economics and outcomes research.
Prior to this, Jens had a distinguished career in the pharmaceutical industry. From 2011-2019, he was the Senior Vice President and Head of Global Access for F. Hoffmann-La Roche based in Basel, Switzerland. Under his leadership, Roche created rapid, broad and sustainable patient access for 12 new medicines and expanded access to essential cancer medicines in lower- and middle-income countries from 29 to 60%. Before this Jens was the Vice President and Head of Global Market Access Primary Care at Pfizer, based in New York and London (2009-2011), Head of Global Pricing & Health Economics at Novartis Pharma in Basel, Switzerland (1999-2009) and Director of Health Economics at the German affiliate of SmithKline Beecham Pharma in Munich (1994-1997).
Laura Pizzi, PharmD, MPH
ISPOR, Lawrence Township, NJ, USA
Dr. Laura Pizzi is Chief Science Officer for ISPOR and Research Professor at Rutgers University. For nearly 25 years, she has led interdisciplinary teams of methodologists, statisticians, and clinicians to design and conduct economic and outcomes analyses on healthcare interventions and is a frequent author, speaker, and mentor on the topic.
At ISPOR, she leads the organization’s scientific strategy and initiatives in collaboration with ISPOR's global members and stakeholders.
Health Preference Research and Value Assessment Frameworks in Digital Health Interventions
This forum delves into the role of patient preference information (PPI) within a value assessment framework (VAF). It will focus on the acceptance of digital health technologies (DHTs) and the patient-centered valuation of patient-facing technologies.
DHTs are revolutionizing healthcare delivery through innovative solutions that improve patient outcomes. However, understanding and integrating patients' preferences pose a significant challenge for the successful valuation and implementation of DHTs.
In this context, patient preferences play an instrumental role in shaping the contours of VAFs. Consequently, these frameworks prioritize outcomes that are directly relevant and meaningful to patients. Furthermore, patient preferences can lead towards greater acceptability and adoption of DH interventions.
Therefore, the seamless incorporation of VAFs into broader health policy strategies becomes essential. A primary obstacle in this process is to strike the delicate balance between swift decision-making processes, the solidity of scientific evidence supporting DH interventions, and the diversity required as DH solutions should cater to a wide array of patient preferences and abilities.
Recognizing that all stakeholders have unique perspectives, success in this field can most likely be achieved through a negotiation process. Consequently, it is of the utmost importance to initially assess patient preferences, develop robust VAFs, and then pinpoint a politically feasible strategy for implementation.
Dr. Mühlbacher, a preference research expert, and Dr. Kolasa, with her multi-stakeholder roles in healthcare and DH experience will spearhead a discussion focused on the elicitation and integration of patient preference data in the valuation of DHT. As an international health policy expert, Dr. Kanavos will share his perspective on VAFs employed to evaluate provider-facing DHTs. Lastly, Dr. Amelung, former president of the German Managed Care Association (BMC) and Professor, Health System Research at the Medical University Hannover will provide a unique perspective on the valuation and implementation of DHTs in Germany
Join the discussion!
Moderators
Axel Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, MV, Germany
Axel Mühlbacher, Dr. rer. oec., is professor of health economics and health care management at Hochschule Neubrandenburg and adjunct professor in the Duke Department of Population Health Sciences, Duke University (USA). He is a CHPIR Senior Research Fellow in the Center for Health Policy and Inequalities Research at the Duke Global Health Institute. His research focuses on methodological advances in decision analysis, operations research, econometrics and choice experiments and their application in the health domain. Recent work includes stated preferences research, empirical research to understand the behavioral aspects of health and problems of multi-criteria decision making to inform the optimal design of policies and regulatory decision making. Past positions include assistant professor at Technische Universität Berlin and research fellow, German Coordinating Agency of Public Health, Albert-Ludwig Universität Freiburg. Axel Mühlbacher holds a M.Sc. in business administration from Eberhard-Karls Universität (Tübingen) and a Ph.D in economics from the Technical University Berlin. From 2010-11 he was a Harkness Fellow in Health Care Policy and Practice. He served as a member of the scientific advisory board of the Institute for Quality Assurance and Transparency in Healthcare (IQTiG) and is chair of the board of the International Academy of Health Preference Research (IAHPR). He currently serves as co-editor of Value in Health, and on the editorial advisory board of PharmacoEconomics, and The Patient – Patient Centered Outcomes Research. Axel Mühlbacher is author of several books and more than 100 peer-reviewed articles. Global Health Institute, Duke University (USA). He serves as a member of the scientific advisory board of the Institute for Quality Assurance and Transparency in Healthcare (IQTiG) and a board member and chair of the International Academy of Health Preference Research (IAHPR). He currently serves as associate editor of Value in Health and on the editorial advisory board of the Journal of Choice Modelling (JOCM), PharmacoEconomics, Int. Journal of Integrated Care (IJIC) and The Patient – Patient Centered Outcomes Research. Axel Mühlbacher is author of several books and more than 100 peer-reviewed articles.
Speakers
Volker Amelung, PhD
Private Institute of Applied Health Service Research (inav GmbH), Hannover, Germany
Prof. Dr. Volker Amelung holds a professorship for International Health Systems Research at the Institute for Epidemiology, Social Medicine and Health Systems Research at Hannover Medical School. Volker Amelung was from 2007 till 2022 the President of the German Managed Care Association (BMC) in Berlin. BMC – a leading platform for health care innovations – elaborates innovative concepts in health care management and policy. In 2011, he founded the Institute for Applied Health research (inav) in Berlin, where he advises national and international companies and conducts studies of applied health services research.
Panos Kanavos, PhD
London School of Economics and Political Science, London, United Kingdom
Dr Panos Kanavos is Associate Professor in International Health Policy in the Department of Health Policy at London School of Economics and Political Science, Deputy Director at LSE Health and Programme Director of the Medical Technology Research Group (MTRG).He has acted as an advisor to a number of international governmental and non-governmental organizations, including the European Commission, the European Parliament, the World Bank, the World Health Organization, the Organization for Economic Co-operation and Development (OECD), and Ministries of Health of over 28 transition, emerging and developing countries.
Katarzyna Kolasa, PhD
Kozminski University, Warsaw, MZ, Poland
Katarzyna Kolasa PhD
Professor of Health Economics at Kozminski University Warsaw Poland
Digital Health Start Me Up Course leader
ISPOR Digital Health SIG founder
“The Digital Transformation of the Healthcare System” Routledge; 1st edition (July 31, 2023)
Driven with passion to improve healthcare, Katarzyna has focused her academic and busi-ness career on the health economics.
She has been working with multiple pricing & reimbursement challenges worldwide for the last 25 years, while holding various Regional and Global leadership positions at Astra Zeneca, BMS, Biogen Idec, Lundbeck, GE Healthcare, Straub Medical, BD and the Swedish county council of Kalmar. Katarzyna is mentor and consultant to start ups involved in the development of innovative medical devices and digital health solutions from both Holland and Poland.
Since 2000 she has been academic teacher and supervisor for over 30 MBA and Phd stu-dents. In partnership with the Polish Medical Research Agency, Deloitte Digital and the Polish Central Hospital of Ministry of Interior Affairs she founded the first Digital Health six months educational program designed for digital transformation leaders working in the healthcare sector. Katarzyna developed innovative Master Program Health Economics & Big Data (HEBDA) which first edition was financed by EU Power Grant 2018 as well.
She is the founder of the Global Special Interest Group Digital Health and short courses “The Role of Digital Endpoints in the Value Generation for Health Technologies”, “Risk-Sharing/Performance-Based Arrangements in Developing Countries” at International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She is the current member of Edu-cational and post member of Health Science Policy Councils at ISPOR as well.
Katarzyna has dedicated her academic research towards the methodological advancements into the value assessment of pharmaceuticals, medical devices, and digital health solutions. Passionate about Big Data, she led the first project of machine learning adaptation for the optimal utilization of CT scanners granted by the Polish Ministry of Health as well. Since January 2022, she is the leader of AI special interest group at the Polish Chamber of Physi-cians. With the patronage of the Polish Parliamentary Commission for Innovation & Digitali-zation, she organized the first dialog about the societal preferences towards the adoption of AI in the healthcare in Poland.
Being coauthor of more than 50 IF publications, she presented her research at more than 60 international scientific conferences. As of 2022, Google Scholar reports over 730 cita-tions to her work.
Educational Symposia
Two concurrent sessions
Advancing Real-World Evidence Generation: Insights from Europe, the US, and China
Digital Conference Pass
Real-world evidence (RWE) plays a pivotal role in healthcare decision making, exerting a profound impact on regulatory approvals, health technology assessment (HTA) evaluations, policy formulation, and clinical guideline development. While RWE holds immense potential in shaping multi-dimensional aspects of healthcare, its implementation and utilization vary across different regions. Understanding regional contexts and strategies is crucial for fostering international research collaboration, accelerating medical innovation, and improving patient outcomes. The presenters will discuss the expanding significance of RWE in healthcare, with examples of experiences and practices observed in the United States (US), France, and China.
Sponsored by Analysis Group
Sponsor
Analysis Group
Moderators
Eric Q. Wu, PhD
Analysis Group, Inc., Boston, MA, USA
Dr. Wu is a health economist with expertise in health economics and outcomes research (HEOR), market access, and scientific evidence strategy. He has conducted research in more than 30 countries on behalf of pharmaceutical and medical device companies, payers, providers, and government agencies. Dr. Wu has contributed to over 200 publications across dozens of therapeutic areas, including regenerative therapies (gene and stem cell), rare and ultra-rare diseases, biologics, and immuno-oncology. Dr. Wu spends a significant portion of his practice developing new scientific methods to address challenges in health care research. He has developed client-focused solutions based on the use of artificial intelligence (AI), medical big data, real-world evidence, and innovative comparative-effectiveness research methodologies.
Speakers
Grégoire Mercier, MD, PhD
Economic Evaluation Unit, CHU Montpellier, Montpellier, France
Dr. Gregoire Mercier is head of Data Science at Montpellier University Hospital and a researcher associated with IDESP (INSERM/University of Montpellier). His work focuses on health data reuse and on health inequalities.
He is co-founder of KanopyMed, a start-up developing digital medical devices and decision support tools for public health. A physician by training, he holds a Master's degree in Health Economics (London Schools of Economics) and a PhD in Economics and Management (University of Montpellier). He was awarded the Commonwealth Fund's Harkness Fellowship in 2018-2019.
Min Yang, MD, PhD
Analysis Group, Inc., Boston, MA, USA
Dr. Yang specializes in health economics and outcomes research (HEOR). She works closely with pharmaceutical, biotech, and device companies to develop HEOR strategies and to generate evidence throughout the product life cycle for value proposition. Dr. Yang designs and conducts studies from pipeline product development through product launch, post-market research, and biosimilar evaluation. She has extensive experience with clinical trial data, health insurance claims databases, electronic medical records, medical charts, primary surveys (including cross-sectional and prospective longitudinal cohort research), and qualitative research for evidence generation, with conventional and innovative methodologies.
Dr. Yang is an expert in clinical outcome assessments – such as patient-reported outcomes (PROs) and clinician-reported outcomes (ClinROs) – as well as health preference research. She has supported the development and validation of multiple PROs and ClinROs, generated evidence for regulatory submissions, and supported real-world evidence (RWE) strategies. A frequent collaborator with academic experts and clinical key opinion leaders, Dr. Yang’s research has been published in many peer-reviewed journals and presented at numerous clinical and economic research conferences. In recognition for her work in women’s health, she received the American College of Obstetricians and Gynecologists (ACOG)/Bayer Healthcare Pharmaceuticals Research Award. Dr. Yang is a licensed oncology surgeon in China and an adjunct assistant professor at The University of Texas at Austin College of Pharmacy. Prior to joining Analysis Group, she was a senior scientist at QualityMetric, Inc.
Jia Zhong, ScD
Analysis Group, Inc., Boston, MA, USA
Dr. Zhong is an epidemiologist who specializes in clinical health economics and outcomes research (HEOR), clinical trials, and large-scale epidemiological studies. She has more than 15 years’ research experience in disease areas such as hematology, pulmonary diseases, diabetes, cardiovascular diseases, oncology, rare diseases, and immunology. Dr. Zhong’s expertise includes comparative efficacy and safety, individualized medicine, predictive modeling, and longitudinal analysis. Her recent work includes leading large-scale prospective cohort studies, evaluating the comparative efficacy of immuno-oncology treatments for regulatory submissions, quantifying direct and indirect resource utilization and costs, and evaluating real-world effectiveness using patient reported outcomes (PROs). Her research has been published in Nature Immunology, Immunity, Proceedings of the National Academy of Sciences, Circulation, Circulation Research, Hypertension, and the International Journal of Epidemiology, among other publications.
Facilitating Change in Obesity: A Multi-Stakeholder Driven Discussion
Digital Conference Pass
The prevalence of obesity (BMI ≥ 30 Kg/m2) is anticipated to rise from 14% to 24% of the population by 2035, affecting nearly two billion adults, adolescents and children worldwide. Obesity is linked to various comorbidities including type-2 diabetes, cardiovascular disease, non-alcoholic fatty liver disease, cancer and others, alongside associations with reduced life expectancy. Globally, obesity is one of the leading causes of mortality, ranking fifth behind high blood pressure, tobacco use, air pollution and high blood sugar, and was responsible for around 8% of all deaths in 2017. In Europe, predictions indicate that 35% of females and 39% of males will be obese by 2035 and that the economic impact in terms of healthcare costs and consequences, and productivity losses will reach over €700 billion. As a complex disease with multifaceted determinants and health consequences, it is unlikely that any single intervention will be able to halt the obesity epidemic. This symposium will explore various perspectives and options for addressing obesity and driving change. To facilitate discussion, we will hear short presentations from a health economist and economic modelling expert, a health technology assessment and policy expert and from a person with obesity. Each will offer a different perspective on how to facilitate change in obesity.
Sponsored by Delta Hat Limited
Sponsor
Delta Hat Limited
Moderators
Zoe Philips, PhD, MSc
Delta Hat Ltd, Nottingham, England, United Kingdom
Zoe is a health economist with over 20 years’ experience in economic evaluation, HTA and health care decision science. She has a PhD in Economics, MSc in Health Economics, and a BSc in Systems Modelling. Having spent her career in industry, academia and consultancy, she has gained extensive experience in HTA, critical appraisal and in communicating health economic and HTA information, as well as in cost effectiveness modelling and statistical analysis. Zoe provides strategic input and impartial advice on health economics, HEOR and HTA projects for industry and also, as a qualified mentor and coach, supports individuals and teams to achieve their goals.
Speakers
Claudia Catacchio, MPhil
ten4 GmbH, Wiesbaden, Germany
Born and raised in Germany in a German/Italian household. Undergraduate studies in the U.S. & France (Psychology / Television, Radio & Film), graduate studies in Cambridge, England. Submission of PhD forthcoming. Specialises in narrative framing of transformative processes. Works in consultancy, mainly on digital transformation processes in large, multinational organisations. Played tennis from age 6 and up to varsity level while at College in the U.S., switched to rowing after a back injury. Represented Cambridge in the Oxford/ Cambridge Boat Race, was British Masters Champion among other titles, and worked as the Head Coach of Newnham College Boat Club in Cambridge for several years. Has been battling obesity for the better part of 25 years until she received an Omega Loop gastric bypass in 2018.
Lars Holger Ehlers, PhD, MSc
Nordic Institute of Health Economics, Aarhus, Denmark
Lars Ehlers, Managing Director, is a former professor of health economics at Aalborg University, Ph.D. and MSc from Aarhus University. Lars has vast experience in research in health economics & health policy, innovative medicine and health technologies, and improvements in healthcare services.
Philip McEwan, PhD
Health Economics and Outcomes Research Ltd., Cardiff, CRF, Great Britain
Phil Is a mathematician and applied statistician and for over 25 years has supported the world’s largest and leading emerging pharmaceutical and biotechnology companies to position their medicines and develop their value and access solutions, to support medicines access
Exhibit Hall Theaters
Multiple 30-minute presentations, Located in the Exhibit Hall
Discussion Groups
Issue Panel on the Identification and Implementation of Clinically Meaningful Thresholds for Clinical Outcome Assessments: Challenges and Considerations
Moderator
Dara O'Neill, PhD
IQVIA, Barcelona, N/A, Spain
Dara is a health researcher and psychometrician with over 15 years’ experience in both academic and professional settings. As part of the Statistics and Psychometrics’ team at IQVIA’s Patient Centred Solutions, she has led diverse projects on the evaluation and modification of clinical outcome assessments. This work has been in particular focussed on patient reported outcome measures and efforts to establish clinical meaningfulness of reported change in interventional settings. Prior to joining IQVIA, Dara was a Senior Research Fellow at University College London, overseeing two separate programs of cross-study data harmonization, including one in support of the UK National Core Studies’ response to COVID-19.
Wouldn't It Be NICE? Could Europe Accept the Consequences of the US Adopting the UK's CEA Framework for Capping Launch Prices?
Moderator
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of stratified medicine for healthcare payers and developers; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A Visiting Professor in Practice at the London School of Economics and a Visting Fellow at the Center for Global Development, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
ISPOR Women in HEOR Session
The Impact of the COVID-19 Pandemic on Gender Distribution of Value in Health Journal Authors
Despite many initiatives to promote and increase representation, women remain underrepresented in the scientific communit 1,2,3 . The COVID-19 pandemic has significantly impacted lives around the world and has presented additional challenges for women 3 . Several studies across various fields of research have reported that the COVID-19 pandemic is associated with a decrease in the proportion of papers submitted for publication or coauthored by women 3,4,5 . T o date, data on the gender of authors of papers submitted to Value in Health ( ViH ) have not been routinely collected and analyzed. The goal of this forum is to discuss the impact of the COVID-19 pandemic on gender distribution of ViH authors . Objectives include exploring whether there is a difference in gender balance of authors for papers published in ViH before (2018 and 2019) and during the COVID-19 pandemic (2020 and 2021) and understanding the characteristics and circumstances that women in HEOR faced regarding publication of research in the United States. The forum will be introduced and moderated by the Women in HEOR co-Lead , Julia Slejko . Nancy Devlin . Sumie Kakehi will present research findings related to gender of authors on ViH submissions and publications before and during the COVID-19 pandemic . and Mike Drummond will discuss the findings from the ViH publication perspective.
References
Thomas EG, et al. Gender disparities in invited commentary authorship in 2459 medical journals. Filardo G, et al. Trends and comparison of female first authorship in high impact medical journals: observational study (1994-2014). Muric G, et al. Gender disparity in the authorship of biomedical research publications during the COVID-19 pandemic. Viglione G. Are women publishing less during the pandemic? Here’s what the data say. Yildirim, T. M., and Eslen‐Ziya, H. The Differential Impact of COVID‐19 on the Work Conditions of Women and Men Academics during the Lockdown.
Moderators
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Julia F. Slejko, PhD is an Associate Professor of Pharmaceutical Health Services Research at the University of Maryland School of Pharmacy and is Co-Director of the Patient-Driven Values in Healthcare Evaluation (PAVE) Center. Dr Slejko’s research is focused on innovative approaches for decision-analytic modeling for economic and health outcomes assessments. She holds a BA in Molecular, Cellular, and Developmental Biology from the University of Colorado Boulder. During her PhD training, she focused on pharmacoeconomics at the University of Colorado School of Pharmacy Center for Pharmaceutical Outcomes Research (CePOR). Her postdoctoral training was completed at the Pharmaceutical Outcomes Research and Policy Program in the University of Washington School of Pharmacy. Prior to her PhD training, she had a 7-year career in drug discovery at Array BioPharma. Dr Slejko serves as a Value in Health Associate Editor, Co-Chair of the ISPOR Faculty Advisor Council, co-lead of ISPOR’s Women in HEOR initiative, and is a member of ISPOR’s Health Science Policy Council.
Speakers
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Nancy is Professor of Health Economics at the University of Melbourne and Senior Fellow at the Office of Health Economics, London. She is Chair of the Board of the EuroQol Research Foundation and served as President of ISPOR (2019-2020). Her principal areas of research expertise are the measurement and valuation of patient reported health outcomes; the cost effectiveness thresholds used in making judgments about value for money; and priority setting in health care. She is currently leading a large programme of research to improve measurement and valuation of quality of life in children (QUOKKA), funded by the Medical Research Future Fund (Australia). Nancy has published >180 original peer reviewed journal papers on a wide range of empirical and theoretical topics in health economics.
Michael Drummond, PhD
University of York, Lichfield, Staffordshire, United Kingdom
Michael Drummond, BSc, MCom, DPhil is professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 700 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Sumie Kakehi, PharmD
Rutgers University, New Brunswick, NJ, USA
Member Group Meetings
Rare Disease Special Interest Group Open Meeting
The Rare Disease SIG invites you to join our Open Meeting where we will provide an overview of our ongoing activities and seek your input on upcoming project ideas and topics. Notably, the SIG is currently focused on a Key Project, titled "Evidence-Based Patient Advocacy in Rare Disease Health Technology Assessment (HTA)." During the meeting, we will present the proposal for this project and discuss opportunities for members to join our Working Group. The SIG will also highlight upcoming events, such as webinars. Your participation and insights are highly valued.
ISPOR Italy-Rome Chapter Meeting
This meeting is open to everyone interested in connecting with the ISPOR Italy-Rome chapter. It offers a valuable opportunity to learn more about the chapter’s initiatives and explore how to participate in the chapter.
12:15 - 12:45
Exhibit Hall Theater
Meeting the Evidence Needs of Regulators, HTAs, and Payers – Optimizing Fit-for-Purpose Real-World Data and Analytics
Digital Conference Pass
Agencies responsible for market authorization and health technology assessment (HTA) around the world are increasingly using real world evidence (RWE) to enhance their decision-making. Life sciences companies must ensure that the evidence they present – both the data and the analytics - will be considered by those agencies to be appropriate and fit-for-purpose.
This HEOR Theater presentation will highlight ways to build confidence that both real-world data and analytics will be considered fit-for-purpose in meeting those varied needs.
Focusing on data requirements for worldwide regulatory pharmacoepidemiology, first we will share lessons learned highlighting the advantages of a data-agnostic approach, informing data decisions with relevant evidence synthesis, understanding regulatory expectations, and obtaining diverse stakeholder insights. Examples will be provided to portray these lessons from multiple perspectives, including submissions targeted to regulators, HTAs and payers.
Audience Objectives:
Appreciate challenges and opportunities related to meeting diverse regulatory and HTA RWE needs and expectations.
Understand how to systematically evaluate RWD sources to meet different regulatory HTA and payer expectations for various uses, such as market authorization, pharmacovigilance and coverage & reimbursement.
Learn how to design RWE studies to meet regulatory and HTA needs with relevant evidence synthesis, stakeholder insights, and other means.
Sponsored by Genesis Research Group
Sponsor
Genesis Research Group
Speaker
Joseph DiCesare, MPH
Genesis Research Group, Hoboken, NJ, USA
Joe DiCesare is President at Genesis Research, an international health economics & outcomes research and real world evidence consultancy. He has over 25 years of experience in HE&OR/RWE, primarily in the pharmaceutical industry.
Prior to moving into his current role at Genesis, Joe was Vice President and Global Head of RWE & Modeling at Allergan. Before joining Allergan, Joe spent 20+ years at Novartis Pharmaceuticals in various leadership roles in HE&OR, Pricing and Access, and Clinical Development. This included leading the Novartis Global and US HE&OR departments, and the Global Oncology Strategic Pricing team.
Joe received his Pharmacy degree from Rutgers University and a Masters in Public Health focused in Epidemiology from the UMDNJ School of Public Health. He is currently an Adjunct Professor at Rutgers College of Pharmacy.
Carole Longson, MBE
Carole Longson Consultant, Manchester, United Kingdom
Carole has a 30-year executive and international board career in life sciences and is a respected international leader in health technology assessment. After a career in drug discovery at GSK, she pioneered NICE’s health technology evaluation programmes as an Executive Director at NICE for 18 years and was Chief Scientific Officer at the Association of the British Pharmaceutical Industry from 2018-2020. She is former President of Health Technology Assessment International and has been a member of the Executive Committee of the European Network in Health Technology Assessment (EUnetHTA) and Scientific Advisory Committees at the European Commission and the WHO. She is currently Vice Chair of the Medicines Discovery Catapult UK and an independent consultant in HTA and market access for the public, private and investment sectors.
Alexandra Sosinsky, ScM
Genesis Research Group, Hoboken, NJ, USA
Alexandra (Allie) Sosinsky designs and implements observational research studies using secondary databases to support regulatory evidence generation needs. She is a trained pharmacoepidemiologist and has experience leading label expansion, external comparator arm, natural history, post-marketing commitment/requirement, post-authorization safety, and meta-analytic studies. She has 12 years of experience in research conduct spanning basic and translational research, clinical trials, prospective observational studies, and retrospective database studies. Allie received her ScM in Epidemiology and Maternal Child Health from the Harvard T.H. Chan School of Public Health, and previously held positions at the Brigham and Women's Hospital Division of Pharmacoepidemiology and the Massachusetts General Hospital Center for Women's Mental Health. Allie has authored over 20 peer-reviewed scientific journal publications on drug development, safety, and regulatory science.
12:15 - 13:15
Special Onsite Join/Renewal Event for ISPOR Membership
Special Onsite Promo! Stop by the ISPOR Booth to join or renew your ISPOR membership! We might just have a surprise for you...
12:45 - 13:15
Exhibit Hall Theater
Trifecta of Willingness to Pay for Rare Diseases: Payers, HCPs, and Society
Digital Conference Pass
Conventional cost-effectiveness analysis may be inappropriate to assess therapies for rare and ultra-rare diseases and further counteract incentives that aim to foster development of such therapies. In this session we will be discussing and exploring alternative evaluation methods that could more appropriately assess the value of interventions for rare and ultra-rare diseases. Sponsored by Lifescience Dynamics
Sponsor
Lifescience Dynamics
Moderators
Sumeet Bakshi, MBBS, MBA
Lifescience Dynamics, London, United Kingdom
Sumeet Bakshi, MBBS, MBA
Senior Vice President
Lifescience Dynamics
Experience
Sumeet is a physician with multi-disciplinary experience working through roles in sales, brand management, strategic planning, business development and research within companies such as Pfizer, J&J and Certara.
Prior to joining Lifescience Dynamics, Sumeet was leading the Real World Evidence practice at Certara where he set up real world research platforms for complex study programs in many therapy areas. Sumeet has consulted for clients on projects in the areas of real-world research, evidence generation strategy, strategic positioning and pricing & reimbursement strategy, amongst others. Sumeet brings strategic thinking, insightful perspectives and strong project/program management skills to the projects that he contributes to.
Education
MBBS from University of Mumbai, Master of Management Studies (Marketing) from University of Mumbai, MBA from Saïd Business School, University of Oxford
Speakers
Thomas J Butt, PhD
BioMarin Europe Ltd, London, United Kingdom
Dr Thomas Butt is Executive Director, Health Economics & Outcomes Research at BioMarin. He is responsible for the global health economics function, developing evidence to demonstrate the value of BioMarin's pipeline and marketed rare disease portfolio. His research interests are in methods of value assessment and he has published 20+ peer reviewed articles in the field. Thomas holds a PhD degree in health economics from University College London (UCL) in the United Kingdom and conducted postdoctoral research at Peking University in Beijing, China.
Domenico Moro, PhD
University of Birmingham, London, United Kingdom
Dr. Domenico Moro is a distinguished economist and educator, serving as a lecturer at the University of Birmingham and an external examiner at the University of Essex. He holds PhDs from The University of Warwick and Università di Bologna. In the private sector, he excels as CEO of Apple Education LTD, focusing on education tours, health economics consultancy, and property management. His rich academic background includes various teaching and research roles, and he has contributed extensively to the field through numerous publications, particularly in health economics and discrete choice experiments.
Andrew Olaye, MSc, PhD
Orchard Therapeutics, London, United Kingdom
Andrew Olaye is the Head of EMEA Market Access at Orchard Therapeutics and has 15 years of experience in pricing and reimbursement, market access, health economics, encompassing various roles of increasing responsibilities at Grunenthal, BioMarin, Vertex Pharmaceuticals and Orchard Therapeutics. Prior to joining the biopharmaceutical industry, Andrew was a Research Fellow at the Centre for BioMedical Sciences, at School of Pharmacy in University of Nottingham, UK. Andrew holds degrees in Pharmacy and health economics, together with a PhD in Regenerative Medicine (Tissue Engineering) from University of Nottingham
13:15 - 14:15
Member Group Meetings
Large Language Models in HEOR
ISPOR Turkey Chapter Meeting
This meeting welcomes all interested in engaging with the ISPOR Turkey chapter. It provides an excellent chance to explore chapter’s activities and discover how you can actively participate.
13:30 - 14:30
ISPOR New Professional Session
The Art and Science of Persuasion: A New Professionals Soft Skills Forum
Graduate school does a great job of teaching technical skills. However, early career success also depends on demonstrating competencies in soft skills like influencing, creative problem-solving, leadership behaviors, critical thinking, cross-functional work, and strategic decision making. This forum will be moderated by a professional development consultant who specializes in soft skills essential for new professionals in the realms of health economics, HTA, RWE, and other life sciences disciplines. This panel-style discussion will include real case studies of early professionals honing these soft skills and applying them to their respective work environments.
Moderators
Ian Bradley, BSc
P31 Academy for Consulting Performance Excellence, Peterborough, CAM, United Kingdom
Ian Bradley is a Partner of P31 Consulting, LLC. Ian has two decades of experience in leadership development, coaching and organisational change, especially in the pharmaceutical and medical device industries. He has worked with major pharma companies, CROs, life science & biotechnology companies and their advisors and partners, in Europe, the US and around the world.
Ian is in demand for his work on communication, persuasion and negotiation, and besides delivering training he is often asked to deliver coaching and leadership interventions for senior teams.
Ian was a Principal with IMS Consulting for three years, where he led and delivered projects in market access and evidence development for global pharma companies. Ian worked early in his career in the Technology Transfer office of a UK university. Following that, Ian’s consulting career included roles with PA Consulting Group (1997-2003, Principal Consultant) and Arthur D Little (2006-2007, Practice Head / UK Healthcare).
Ian has a BSc(Hons) from the University of Durham in the UK in Engineering and Management and is based near Cambridge in the UK.
Speakers
Ka Keat Lim, MPharm, MSc, PhD
Kings' College London, London , LON, United Kingdom
Ka Keat Lim is a health economist currently working as a research fellow at King's College London (KCL), UK. At work, he works mainly on systematic reviews, longitudinal data analyses and economic evaluations, with particular interests on precision medicine, prevention and screening interventions. He is also the Co-Chair of Early Career Researchers & Teachers Network (ECRTN) at KCL School of Life Course & Population Sciences. Outside of work, he is a Member of the ISPOR New Professionals Committee, and Chair-Elect of ISPOR Precision Medicine & Advanced Therapy SIG. He enjoys reading non-fictions, listening to podcasts and strolling in the park during his free time.
Kate Noble
Remap Consulting, Macclesfield, CHW, United Kingdom
Kate has over 10 years of experience in business development, including business-to-business sales, consultancy provision and client relationship management across pharmaceutical and biotech industries. Kate has a keen eye for new and innovative commercial opportunities worldwide and works strategically with both internal and external stakeholders.
Kate’s passion lies in her strategic ability to manage stakeholders, perform commercial data analysis, project sponsorship and delivery of complex recommendations.
Riku Ota, MPH
Novo Nordisk Pharma Ltd., Chiyoda, Tokyo, Japan
Riku Ota is a motivated public health professional with progressive experience in the pharmaceutical industry and consultancy in Asia Pacific and Europe. His career background started in the clinical development of innovative medicines for inflammatory bowel diseases (IBD). His current role in Novo Nordisk Pharma Ltd. (Japan) is in the Market Access and Health Economics Outcome Research (HEOR) to secure broader healthcare access for patients by demonstrating the clinical and economic values of new health technology as a HEOR and MarketAccess Manager. He is an active member of the Health Equity Special Interest Group (SIG) of ISPOR in Asia-Pacific. He is also nominated as a New Professional Steering Committee member for the 2023 to 2024 term at the ISPOR. He acts as an HTA Taskforce Lead in the EFPIA Japan Access Committee, which is one of the largest pharmaceutical industry bodies. He holds a BSc in Statistics from the University of Otago, New Zealand, and an MPH from King's College London, the United Kingdom. His primary interests are global public health and health disparities, primarily due to socioeconomic factors. He is pursuing a Master of Business Administration (hybrid part-time) at the IE Business School.
13:45 - 14:45
Breakouts: IP, WS, & OBS
How to Determine Unmet Needs for More Needs-Driven Product Development and Healthcare Decision-Making?
Digital Conference Pass
Level: Intermediate
ISSUE: Identifying unmet medical needs is essential for needs-driven product development and healthcare decision-making. Although agreement exists on the importance of focusing research and development on unmet needs, varying interpretations exist towards the meaning and concept of unmet needs, and their identification and application in product evaluation and healthcare decision-making. This session discusses, through a multi-stakeholder lens, challenges and opportunities for unmet need identification and structured assessment in decision-making.
OVERVIEW: This session will highlight insights from different stakeholders (Policy, HTA, academia, patient) on i) the concept of unmet need (distinguishing between patient and societal need), ii) the identification of unmet needs, and iii) avenues for their more systematic integration and assessment in healthcare decision-making, highlighting current and novel methodological approaches and case studies. Opportunities and challenges will be discussed in view of the new European pharmaceutical legislation where the concept of ‘Unmet medical needs’ is centrally embedded. A concrete case example on unmet needs identification, which is currently under development by the Belgian Health Care Knowledge Centre (KCE) in collaboration with academia, will be showcased. Furthermore, the session will delve into unmet need determination in the context of rare diseases, highlighting specific challenges, methodological considerations, and insights from the patient perspective. Speakers will address the current status and challenges, and highlight novel methodological approaches and learnings derived from concrete case studies. Panellists will have 10 min presentation time. 25 minutes are foreseen for discussion during which attendees’ opinions will be probed using the ISPOR polling tool. Multiple stakeholder groups will benefit from attending this issue panel including policymakers, HTA assessors, payers, regulators, patient representatives, academics, pharmaceutical industry, and market access representatives.
NOTE: There will be a discussion group on this topic following the session. Join us in the Discussion Lounge in Hall E North from 15:15-16:15 in Discussion Group A.
Moderators
Isabelle Huys, PharmD, PhD
Department of Clinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, VBR, Belgium
Prof. dr. Isabelle Huys is full professor at the University of Leuven (Belgium). She leads the research group on Regulatory Sciences at the Faculty of Pharmaceutical Sciences, embedding the Patient Evidence and Preference Center (PPRC). Her research focusses on market access of medicines and therapies, with particular attention to the inclusion of the voice of the patient in drug development, evaluation, market access and usage. In addition, ethical, legal and social issues (ELSI) are studied in an interdisciplinary context.
Panelists
Liese Barbier, PhD, PharmD
Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, VBR, Belgium
Liese Barbier, PharmD, PhD, is a postdoctoral researcher at the KU Leuven in Belgium, in the Regulatory Sciences & Pharmaco-Economics research unit. Her research interests and activities focus on regulatory, clinical and policy aspects of medicinal products, including clinical drug development, regulatory evaluation, market access policies, and clinical product implementation. She has a particular interest in optimizing regulatory and decision-making frameworks for complex medicines and in areas with high unmet medical need. Her PhD research focused on regulatory, clinical and policy challenges related to biosimilar market entry in Europe, through which multi-stakeholder informed recommendations were generated to foster sustainable off-patent biological market dynamics. In 2019, Liese was seconded to the European Medicines Agency as National Expert. Here, she was part of the Oncology, Haematology and Diagnostics office of the Human Medicines Evaluation Division and focused as Product Lead on the evaluation of biosimilar candidates in oncology. Further, she provided scientific input to biosimilar related initiatives across the Agency and coordinated as Scientific Lead the Biosimilar Medicinal Products Working Party.
Francois Houyez, Patient Advocate
Rare Diseases Europe, Paris, France
François Houÿez is Director of Treatment Information and Access at the European Organisation for Rare Diseases EURORDIS.
He has always been working as a patient advocate since the early 90s, first in the HIV/AIDS advocacy, and in rare diseases since 2003.
His experience with compassionate use programmes started in 1988.
He pioneered patient advocacy with the European Medicines Agency as part of the first patients’ delegation that engaged dialogue with the Agency back in 1996.
François is also a patient.
Muriel Levy, PhD
KCE, Bruxelles, Belgium
Muriel Levy is an expert in health economic analysis at the Belgian Healthcare Knowledge Centre (KCE) since 2022. She is currently involved in the identification and measurement of unmet needs (NEED project) and in the new edition of Belgium's health system performance assessment. Before working at KCE, she worked as a researcher in healthcare management at Ghent University and as a consultant for the WHO and the OECD, covering a range of topics (e.g. mental health, health system resilience, health information systems). Muriel Levy holds a PhD in Population Health and a MSc in Global Health Science and Epidemiology from the University of Oxford, and a BSc in Economics from LSE.
Setting up a Harmonized Methodology to Promote Uptake of Early Feasibility Studies for Clinical and Innovation Excellence in the European Union: What Value(s)?
Digital Conference Pass
Level: Intermediate
ISSUE: Nowadays, in the EU there are no standardized procedural frameworks for the uptake of EFS. This hinders the application of the lifecycle approach for clinical evidence generation of MDs envisaged by EU Regulations (MDR, IVDR, HTAR), slows down European patients’ access to innovation, and EU competitiveness. The HEU-EFS Project is a 4 year project funded by the Innovation Health Initiative and 6 industry partners, which aims at addressing these challenges by developing a harmonised methodology for the uptake of EFS in the EU.
OVERVIEW:
Prof. Callea (15 minutes) will present the HEU-EFS Project rationale and objectives, and will discuss with panelists the potentials of the future EU EFS Program. Prof. Melvin (10 minutes) will discuss the relevance of the EU EFS Program for the public entities involved in the assessment of medical technologies and the expected impact on the current regulatory framework as well as on the clinical evidence-generation process in the EU. Prof. Kidholm (10 minutes) will present the advantages and challenges perceived by public teaching hospitals regarding the establishment of an EU-EFS Program. Mr. Rappagliosi (10 minutes) will discuss the expectations of developers of breakthrough technologies, also given previous experience in the conduction of EFS in the USA. He will explain the importance for companies to access a clear framework to uptake EFS in the EU and the importance of public-private collaboration for its development. In the last 15 minutes, Prof. Callea will collect questions from the audience to be discussed with panelists in a round table. A number of stakeholders will benefit from attending the panel session including EU-wide and national regulators, competent authorities, notified bodies, HTA bodies, healthcare providers, patient organizations, research organizations, legal and ethical experts, CROs, and health technology developers.
Moderators
Giuditta Callea, PhD
SDA Bocconi School of Management, ROMA, RM, Italy
Giuditta Callea is an Associate Professor of Practice at SDA Bocconi School of Management. Her main areas of research include health economics and health policy with a special focus on lifecycle generation of clinical evidence, funding & reimbursement and public procurement of new medical technologies, determinants of diffusion of new medical technologies, and economic evaluation of healthcare technologies. She is a member of ISPOR. She is the author of several publications in health economics, Topic Editor of Clinical Therapeutics - area Pharmacoeconomics, Outcomes, and Health Policy, and reviewer for several international academic journals.
Panelists
Kristian Kidholm, PhD
Odense University Hospital, Odense, Fyn, Denmark
Kristian Kidholm is a PhD, professor and head of research at CIMT - Center for Innovative Medical Technology at Odense University Hospital. CIMT is a leading center for research in digital health technology in Europe. Kristian research is focused on assessment of the value of digital health service and economic and organisational studies of digital health technologies.
Tom Melvin, PhD
Trinity College Dublin, Dublin, Ireland
Tom Melvin is Associate Professor of Medical Device Regulatory Affairs in Trinity College Dublin. Prior to this, Tom worked as a senior medical officer in medical devices at the Health Products Regulatory Authority, where he served for 7 years.
Tom Co-chaired the Clinical Investigation and Evaluation Working Group of the European Commission for 5 years. Tom has led various taskforces, work packages and projects at national and international level on regulatory and scientific topics related to medical device regulation.
Tom is a member of the International Rare Disease Research Consortium (IRDiRC) working group on MedTech for rare diseases, and a member of the National Research Ethics Committee for Medical Devices in Ireland. Before working in medical devices, Tom worked as a medical doctor for a number of years.
Fanny Van Der Loo
Edwards Lifesciences, Brussels, Belgium
Early Endpoints in Oncology: Increasingly Common in Clinical Trials Yet Frequently Challenged in Health Technology Appraisals (HTAs)
Digital Conference Pass
Level: Intermediate
ISSUE: In recent years, more and more regulatory approvals in oncology are based on improvements in early endpoints (e.g. progression free survival, response rate) with the goal of bringing new innovative treatments faster to cancer patients. However, early endpoints are frequently challenged in HTAs with continued preferences for final endpoints (overall survival) or certainty of benefit on final endpoints either through long-term follow up or validation of the early endpoints as a surrogate for overall survival. This often leads to delays in access and reimbursement of new oncology drugs. Meanwhile, the evidence required for surrogate endpoints validation varies across HTA bodies and the success in surrogate endpoint validation in oncology is rare.
OVERVIEW: Dr. Sasane will introduce the issue and panelists (5 min). Ms Zheng will represent the drug developer’s perspective on the rationale of using early endpoints as primary efficacy endpoints in oncology trials, including changing treatment landscape, feasibility challenges of using overall survival as the sole primary endpoint, value of early access to innovative therapy, the patient-relevance of early endpoints and the practical challenges of surrogate endpoint validation (13 min). Dr. Latimer will share the HTA perspective on key concerns of making HTA recommendations based on early endpoints with immature OS data and where surrogate endpoint validation may be insufficient to address reimbursement questions (13 min). Ms. Cope will provide her research perspective and propose a stepwise framework to assess the feasibility of surrogacy meta-analysis to ensure underlying assumptions are explored systematically in anticipation of HTA requirements (14 min). There will be 15 mins to address audience questions and debates. The panel hopes to benefit the three types of stakeholders represented by the panelists to facilitate future policy and research initiatives to improve the understanding and robustness of early endpoints in reimbursement decision-making.
Moderators
Kyle Hvidsten, MPH
Sanofi, Cambridge, MA, USA
Kyle Hvidsten is Head of the Global Specialty Care Health Economics and Value Assessment department. In this role, Kyle oversees input into clinical trial design and the development of economic models and RWE studies to support scientific exchanges with health technology assessment agencies and healthcare payer organizations across the globe. Prior to joining Sanofi, Kyle held several positions in US and Global health economics and outcomes research with increasing levels of responsibility over the past 20+ years. He holds a MPH degree from Columbia University and a BA from Emory University.
Panelists
Shannon Cope, MSc
PRECISIONheor, Vancouver, BC, Canada
Shannon Cope is a Senior Vice President with PRECISIONheor with fifteen years of experience consulting for biopharmaceutical industry. Shannon leads evidence synthesis projects involving (network) meta-analyses, population-adjusted indirect comparisons, multi-level network meta-regressions, surrogacy, and economic models. Her publications relate to application and extension of evidence synthesis methods related to (network) meta-analysis, the feasibility assessment process, time-to-event outcomes, and the integration of individual patient data from clinical trials, real-world evidence, and expert opinion. She has an MSc in Health Administration from the University of Toronto, Canada.
Nicholas Latimer, MSc, PhD
The University of Sheffield, Sheffield, DBY, Great Britain
Nick is a Professor of Health Economics at the University of Sheffield, a Yorkshire Cancer Research Senior Fellow, and an Analyst for Delta Hat Ltd. He was a member of NICE Technology Appraisal Committee B for 5 years and is a member of the NICE Decision Support Unit (DSU). Nick’s research focuses on survival analysis in economic evaluations. He has authored four DSU technical support documents related to survival analysis, treatment switching, and partitioned survival models. His current work involves investigating the use of cancer registry datasets to estimate the comparative effectiveness of cancer treatments used in clinical practice.
Medha Sasane, MHSA, MS
Sanofi, Cambridge, MA, USA
Ms Zheng is currently Senior Director of Global Health Economics and Value Assessment in Sanofi. She has over 12 years of experience in the field of HEOR with a strong focus in oncology. Ms Zheng works in the pharmaceutical industry for more than 7 years and has taken on different global and US focused roles on launched products and pipeline assets.
Use of External Control Arms in Rare Disease: Are We Moving Towards an International Gold Standard and How Can We Facilitate Progress?
Digital Conference Pass
Level: Intermediate
ISSUE: Historically, rare diseases (RD) have faced challenges generating gold-standard evidence through randomized controlled trials (RCT) due to limited population sizes and ethical issues. External control arms (ECA) emerged as an alternative and now play an important role in market authorization and reimbursement decisions for RD products.
While ECAs in RD have been accepted by the EMA as pivotal and sole comparative evidence instead of RCT, European HTA bodies and payers have proven more reluctant, arguing that evidence from ECAs present too much uncertainty for decision-making. This resulted in treatment access being delayed or denied. Despite the availability of case-by-case joint scientific advice and recent publications related to the generation of robust alternative evidence to RCTs, transparent eligibility criteria for ECA are still missing, preventing manufacturers from anticipating their acceptability. Constructive discussions between stakeholders to determine practical conditions of use for ECAs are thus necessary to pave a path forwards.
OVERVIEW: NICE was the first European HTA body to provide guidance for conducting ECAs in its 2022 RWE framework. HAS and CADTH released their recommendations in 2023 which shared conclusion as NICE. In parallel, the FDA released a draft guidance on ECAs while the EMA published a reflection paper on the use of single arm trials (SAT).
Jasdeep Hayre will kick-off the discussion by detailing the NICE position on ECA. Andrew Thomson, representing the EMA, will present the SAT reflection paper and its commonalities/differences with HTA bodies’ approaches. Finally, Amr Makady will represent the industry viewpoint and discuss the operational and strategic applicability of those guidances. The discussion will be moderated by Carla Vossen who will identify areas of alignment/divergence across all parties and steer the discussion on the impact of the future EUnetHTA assessment on the global development of RD drugs and the continued challenge of generating internationally harmonized fit-for-purpose evidence.
Moderators
Carla Vossen, PhD
Syneos Health, Amsterdam, Netherlands
Carla Vossen, PhD, is a Senior Director Epidemiology, RWE at Syneos Health.
Dr. Vossen has over 20 years of experience in epidemiological research as a clinical epidemiologist in academia and as an HEOR/epi/HTA consultant for the pharmaceutical industry. She has experience in observational studies from start to end, including design of externally controlled studies. Therapeutic and methodological topics of interest include innovative observational study designs, rare disease, oncology and genetics/genomics.
Panelists
Jasdeep Hayre, BSc MSc
National Institute for Health and Care Excellence, Manchester, United Kingdom
Jasdeep Hayre is an Associate Director within the England’s National Institute for Health and Care Excellence (NICE). He currently provides leadership for the health technology assessment of new medicines at NICE. Within his role he is supporting the development of new methods and processes for a new proportionate approach to technology appraisals (PATT). Jasdeep has worked at NICE since 2011 and previously worked on the methodology and quality assurance of economic evaluations in guidelines and health care staffing levels. He holds a post-graduate degree in Health Economics from the University of York, and a degree in Economics from the University of Nottingham.
Amr Makady, PharmD, PhD
Janssen-Cilag BV, Breda, Netherlands
Amr Makady is a doctor in pharmacy currently working on new business development and early product evidence generation plans for European market access at Janssen. He previously worked at the ZIN (Dutch HTA agency) for 6 years where he specialized in RWE use (IMI Get Real project) and accelerated access. Amr holds a PhD in RWE use for HTA.
Patrice Verpillat, MD, MPH, PhD
European Medicines Agency, Amsterdam, Netherlands
Dr. Patrice Verpillat is the Head of the Real World Evidence (RWE) Workstream at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. He has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) in order to bring RWE into research, access and life-cycle product management.
Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (efpia) and ISPE.
Incorporating External Evidence Into Extrapolations: A Debate on the Trade-Offs Between Complexity and Simplicity
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Level: Intermediate
ISSUE: Producing plausible lifetime extrapolations is fundamental to appropriate decision-making within health technology assessment (HTA). However, the pipeline of novel therapies relying on surrogate outcomes and immature survival data continues to grow, creating challenges for modelers estimating lifetime outcomes for HTA. These challenges have led to an increase in proposed methods designed to improve extrapolation using external data such as real-world evidence (RWE) and expert elicitation. While HTA entities such as the National Institute for Health and Care Excellence (NICE) have endorsed the use of RWE and expert elicitation, modelers are left with multiple choices and little guidance on when and how these methods may be used in practice. Furthermore, the use of increasingly complex methods combined with a lack of research comparing performance across methods may lead to further scepticism of HTA results.
OVERVIEW: This panel will discuss the trade-offs between the benefits gained through the use of advanced extrapolation methods leveraging external evidence, and simplistic and transparent methods for HTA decision making. R. Brett McQueen will moderate and review the issue based on the current state of the literature. Bart Heeg will represent the perspective of a methods developer in the survival extrapolation field and articulate reasons for using more advanced methods. Dawn Lee will represent the perspective of a model reviewer for NICE submissions and articulate the problems that increasingly complexity causes, the benefits of keeping things simple where possible and the need for transparency and consideration of the manner in which data is presented in survival extrapolation methods. Finally, Ash Bullement will present the perspective of an advisor and consultant developing submissions to HTA agencies on behalf of manufacturers. All three panellists will offer contrasting perspectives on the trade-offs between simplicity and complexity of survival extrapolations for HTA decision making.
Moderators
Robert McQueen, PhD
University of Colorado, Denver, CO, USA
R. Brett McQueen is an Associate Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
Panelists
Ash Bullement, MSc
Delta Hat, Nottingham, NTT, United Kingdom
Ash is an Analyst at Delta Hat, a UK-based HEOR consultancy, and is also a part-time PhD student at ScHARR, University of Sheffield. Ash's PhD is focused on methods for survival extrapolation that make use of external evidence, particularly in the context of cancer treatments
Bart Heeg, MSc, PhD
Cytel Inc., Rotterdam, ZH, Netherlands
Bart is Vice President HEOR at CYTEL. Bart has a PhD in health economics. He has over 20 years of experience in heath economic and outcomes consulting. He focusses on indirect treatment comparisons, surrogacy analyses and health economic modelling.
Dawn Lee, MMath, MSc
University of Exeter, Exeter, DEV, United Kingdom
Dawn is an Associate Professor of Health Economics and Health Policy, working with the Peninsula Technology Assessment Group (PenTAG), one of 11 research units in the UK providing expert advice on the clinical and cost-effectiveness of new drugs to the National Institute of Health and Care Excellence (NICE). She is also an existing member of the NICE interventional procedures appraisal committee.
Dawn joined PenTAG / Exeter University in Sept 2022 following over 15 years in economic consultancy where she worked most recent as the Chief Scientific Officer for a medium sized health economics consultancy (Lumanity; formerly BresMed). She is a health economic modeler who has conducted over 50 UK Health Technology Assessment submissions and worked in over 30 countries globally, a member of NICE’s interventional procedures advisory committee (IPAC) and the R for HTA group. Dawn’s key project achievements include representing manufacturers at ~30 HTA Committee meetings, working on the first ever immune-oncology submission to NICE (TA268), first ever PD-1 submission to NICE (TA384) and a considerable number following this and the first ITC accepted by G-BA.
Dawn’s main research interests are oncology modelling; particularly immune-oncology and therefore flexible survival modelling and the incorporation of external data within extrapolations, structured expert elicitation and improving diagnostic pathways.
Dawn is currently working on NICE's pathways pilot.
The "Population, Intervention, Comparator, and Outcome" Puzzle: Navigating Uncertainty in the Joint Clinical Assessment Era
Digital Conference Pass
Level: Intermediate
PURPOSE: The European Joint Clinical Assessment (JCA) will provide a single process for the Health Technology Assessment (HTA) for member states (MS). The JCA will be informed by a ‘Population, Intervention, Comparator, Outcome’ (PICO) survey which may result in multiple PICOs within a single submission. Although the JCA is intended to expedite patient access for innovative therapies, the need to collect and present data for multiple MS may create challenges to traditional HEOR. This workshop will explore these challenges using case studies, and possible solutions using online tools for situations where PICOs diverge between MS.
DESCRIPTION: The session will be introduced by Prof. Wim Goettsch who will describe the JCA opportunities and challenges for patients, manufacturers and HTA; and the potential high-stakes of establishing PICOs (7 minutes). Maria Rizzo will review an oncology case study identifying risks of divergent PICOs, driven by varying standards of care, and how an online, all-encompassing comparative effectiveness framework may help prepare evidence generation activities (15 minutes). A case study on reporting considerations using an online systematic literature review tool that generates automated reporting by PICO filtering will be presented by Maria Rizzo (8 minutes). Andrew Satherley will then present a case study on anticipating MS PICOs using an online survey tool to elicit MS HTA and clinical expert PICO requirements, before consolidating PICOs (10 minutes). Finally, leaders will discuss implications for anticipating the JCA scope in the HEOR strategy (5 minutes).
There will be interactive participation; the audience will be asked to make key decisions on their HEOR planning based on the case studies presented. The audience will be asked to consider how tools and other strategies can help navigate any uncertainty in the JCA era (15 minutes).
Moderators
Wim Goettsch, PhD
National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He has serves as a special HTA advisor to Zorginstituut Nederland and a former director of EUnetHTA JA3 Directorate.
Discussants
Maria Rizzo, MSc
Cytel, London, London, United Kingdom
Maria Rizzo is a Vice President at Cytel with over 15+ years of relevant experience in evidence synthesis. Maria started her career conducting SLRs to inform clinical guidelines in mental health for the National Institute for Health and Care Excellence. She then spent over 12 years working in consultancy to support pharmaceutical companies navigate the reimbursement pathway. Maria specializes in considerations on PICOS selection to match the treatment pathway for reimbursement application.
Andrew Satherley, MSc
Lightning API, Lincoln, LIN, United Kingdom
Andrew is Joint Managing Director at Lightning Health, where he is specialised in supporting the evidence generation, value demonstration and HTA strategy development for innovative medicines. He has extensive experience in Health Economics and Global Market Access, including multiple roles in Health Policy Research and Market Access Consulting.
Andrew obtained his MSc Health Economics from the University of York, and has led a policy review at the Nova School of Business and Economics, Lisbon, regarding European Commission incentives for the development of orphan drugs
Every Patient Matters: Introduction to Multi-Dimensional Thresholding in Health Preference Research
Digital Conference Pass
Level: Introductory
PURPOSE:
Patient preferences are routinely used to inform decisions throughout medical product development, such as regulatory approval and reimbursement. While discrete choice experiments have become the gold standard for preference elicitation, they suffer from low precisions in small samples and may fail at the individual level. This limits their application in rare diseases and in measuring preference heterogeneity in smaller subgroups. To overcome these limitations, multi-dimensional thresholding was developed as part of the European Medicines Agency (EMA) study on treatment preferences of patients with multiple myeloma. This workshop introduces the audience to the use of multi-dimensional thresholding in health preference research using the EMA patient preference study and an interactive exercise. DESCRIPTION:
Dr Ho will open the workshop by briefly discussing how patient preference information is used throughout medical product development. Dr Postmus will introduce the EMA multiple myeloma study and discuss example uses of thresholding technique for preference elicitation at the EMA and the US Food and Drug Administration (FDA). Dr Heidenreich will present on how to design multi-dimensional thresholding instruments and demonstrate their robustness with results from computational experiments. Dr Tervonen will discuss data analysis and conduct a live preference elicitation with workshop participants using their mobile devices. The results will be presented during the session and compared with results of N=560 multiple myeloma patients from the EMA study. HEOR and regulatory affairs scientists benefit from attending this introductory workshop.
Discussion Leaders
Kerrie-Anne Ho, PhD
UCB Pharma, Slough, SLG, United Kingdom
Kerrie-Anne Ho, PhD, is a Patient Preference Lead at UCB. During her tenure, she has been impactful in raising the awareness of the value of patient preferences in pharmaceutical drug development, providing strategic guidance and leading the implementation of meaningful patient preference research. Kerrie-Anne brings over 10 years of experience from a diverse life sciences background in various consulting and clinical research roles in the UK and Australia.
Discussants
Sebastian Heidenreich, PhD
Evidera, London, LON, United Kingdom
Douwe Postmus, PhD
University of Groningen, University Medical Center Groningen, Groningen, GR, Netherlands
Dr. Douwe Postmus is working as an assistant professor at the Department of Epidemiology of the University Medical Center Groningen, The Netherlands. His research focuses on applying statistical and decision analysis techniques to decision making problems in health care, including regulatory, reimbursement, and shared decision making problems. He has an h-index of 26, co-authored 78 publication(s) receiving 1703 citation(s).
Tommi Tervonen, PhD
Kielo Research, Zug, ZG, Switzerland
Dr Tervonen is the Chief Scientist of Kielo Research and key opinion leader in patient preferences and benefit-risk assessment. His expertise influences decision maker opinion and he has been consulted by agencies such as the US Food and Drug Administration (FDA) and UK National Institute of Health and Care Excellence (NICE). Dr Tervonen co-chairs the ISPOR Task Force on Good Practice in Quantitative Benefit-Risk Assessment and has served as principal investigator in dozens of patient preference studies, including an IMI PREFER industry case study, and studies used in regulatory filings.
Podium Sessions
The Role of Artificial Intelligence: From Economic Modelling to Clinical Decision Making
Digital Conference Pass
Artificial Intelligence (AI) is a quickly developing approach searching its appropriate role in both medical research and clinical medicine. This session gives an overview on how AI currently can support HTA from economics modelling to clinical decision making.
Moderator
Erwin De Cock, MSc
Syneos Health, Barcelona, Spain
Erwin is an Executive Director, Real World and Late Phase at Syneos Health. He is an industry expert in the design of Time and Motion (T&M) observational studies that aim to quantify efficiency in healthcare delivery and currently focuses on feasibility assessments and implementation strategies for chart abstraction and contemporaneous data collection to build external control arms. Mr. De Cock has a Degree in Applied Economics from Katholieke Universiteit Leuven (Belgium) and has worked as a health economist and real-world evidence scientist for over 20 years.
P40: Man Versus Machine: Can AI-Assisted Technology be Used to Support the Development of Economic Models?
2:30PM - 2:45PM
Medland S 1 , Davies H2 , Butler K3 , Moss J2 1 York Health Economics Consortium Ltd, Cardiff, CRF, UK, 2 York Health Economics Consortium Ltd, York, York, UK, 3 York Health Economics Consortium Ltd, York, YOR, UK
OBJECTIVES: This research aims to explore whether an artificial intelligence assisted technology (AI) can be utilized for writing and reviewing standardized functions in Visual Basic for Applications (VBA) for health economic models (HEM) built in Microsoft Excel.
METHODS: A fully incremental analysis (FIA) is required for health technology evaluations submitted to NICE to determine whether technologies are dominated or extendedly dominated. An AI was asked to write a VBA function to automate an FIA for a HEM. Feedback was provided to the AI and new code was generated three times. Each iteration was timed, reviewed and results cross-checked with an example model. Additionally, a modeler created a bespoke VBA function to automate an FIA. The AI was asked to review the code and make it more efficient. The changes made to the code by the AI were reviewed by a modeler and results were validated.
RESULTS: The AI required a specific research question to ensure the function returned the appropriate outcomes. For each iteration, the code and accompanying guidance was generated in under two minutes. Minor issues with the code included use of hardcoded values; missing arguments; incorrectly defined variables; use of key words not used in VBA (e.g. continue for), and outputting results in an incorrect format. Two iterations correctly determined dominance, but extended dominance calculations were incorrect in all iterations. The third iteration contained a calculation error causing incorrect results. When asked to review a modeler’s function, the AI suggested several valid adjustments; however, the new code was poorly styled, and some key calculations were incorrect.
CONCLUSIONS: Code generated by AI may improve in future if more opensource model code is published. Currently, AI cannot replace the expertise and experience of modelers but has the potential to be a useful tool to review code and give advice.
P39: Machine Learning to Identify Atopic Dermatitis Prevalence Using Healthcare Utilisation Patterns of Both Diagnosed and Non-Diagnosed AD Patients Based on Danish Register Data
2:15PM - 2:30PM
Liljendahl M 1 , Torpet M2 , Lyngsie PJ3 , Rudolfsen JH4 , Pedersen M5 , Ibler KS6 1 Herlev and Gentofte Hospital, Copenhagen, 84, Denmark, 2 Sanofi, Copenhagen Oe, Denmark, 3 Sanofi, Copenhagen, Denmark, 4 EY, Copenhagen, Denmark, 5 EY, Frederiksberg, Denmark, 6 Zealand University Roskilde, Roskilde, Denmark
OBJECTIVES: Atopic Dermatitis (AD) is a chronic skin disease, causing itch and recurrent eczematous lesions. In Danish national register data, adults can only be identified as AD patients if they have hospital diagnosed AD. The purpose of this study was to develop an algorithm to identify AD patients by proxy; using contacts with primary care, prescription medication, and hospital contacts not related to skin disease.
METHODS: Individuals fulfilling a prescription for dermatological preparations between 1996 – 2022 was extracted as potential AD patients. Individuals were classified by hospital diagnosis as ‘AD’ (ICD-10: L20), ‘No AD’ (ICD-10: indicating other skin disease), or ‘Uncertain AD status’ (no hospital diagnosis). ‘AD’ and ‘No AD’ patients (n = 22,772) were used to develop the algorithm. All use of healthcare services two years prior to hospital diagnosis was used as potential predictors (~11,450 variables). The data was split into train/validation set (70/30). Recursive feature elimination were performed, using criteria of mean decrease in accuracy in a random forest model. The best model with ≤25 features was selected. Spurious predictors were removed based on a clinical relevance. The model was evaluated on the validation set, before applied to the patients with uncertain AD status.
RESULTS: From 1996 – 2022, 570,094 individuals had uncertain AD status. Most important predictors were corticosteroid medication for dermatologic use, consultations with dermatologist and age. The internal model accuracy was 85% (AUC: 0.82, Sensitivity: 0.78, Specificity 0.87). The fit measure in the validation set showed no substantial optimism with an accuracy of 81% (AUC: 0.79, Sensitivity: 0.72, Specificity: 0.85). In total, 255,067 were predicted to have AD in the study period, or an incidence rate of non-hospital diagnosed AD of 158/10,000 adults in Denmark.
CONCLUSIONS: Healthcare utilization patterns from Danish register data can identify patients with AD from patients with other dermatologic diseases.
P37: Can Artificial Intelligence Methods Improve Mapping of Preference Based Measures? an Application of Symbolic Regression
1:45PM - 2:00PM
Crott R
OBJECTIVES: Many mapping algorithms have been published using a variety of traditional statistical methods. This research aims at comparing mapping from Symbolic Regression (SR) approach with published mapping algorithms from standard approaches. SR is an established technique for identifying the optimal’ mathematical expressions that can describe relationships within a data structure .
METHODS: We retrieved the individual patient data from two previously published papers mapping the EORTC QLQ-C30 to the EQ-5D-3L in Non-Small Cell Lung Cancer patients (Jang 2010, Crott 2018, Khan & Morris 2014). Using a simulated annealing-based SR approach we searched for a best fitting model arithmetic function, including also trigonometric functions to explore the combinatorial regression equation space using the full data sample. The best fitting equation was identified by minimizing the RMSE, without limits to the equation complexity score. Goodness-of-fit (GOF) was further checked by MAE and R² and compared with that of the traditional previously published results. All searches were performed with the TuringBot software version 2.16.1.
RESULTS: very different best-fitting equations following simulated annealing optimization were obtained compared to the published ones for the Jang, TOPICAL and SOCCAR data. For the Jang data a small improvement of GOF of about 5%-6% was obtained, although depending on the GOF measure, with no RMSE below 0.17 while for the TOPICAL and SOCCAR data the SR performed similar or slightly worse than OLS.( RMSE 0.1842 vs 0.183 and 0.148 vs 0.141 respectively).
CONCLUSIONS: Using Symbolic Regression either only slightly improved the GOF of the mapping algorithms compared to more traditional regression methods This may be due to the multimodal distribution features of the EQ-5D-3L and supports the use of 2 or 3-part models for mapping to the EQ-5D_3L
P38: Forecasting Emergency Department Waiting Times Using Deep Neural Networks
2:00PM - 2:15PM
Pak A 1 , Trinh K2 1 The University of Queensland, St Lucia, QLD, Australia, 2 CSIRO, Melbourne, VIC, Australia
OBJECTIVES:
To deliver accurate ED waiting times forecasts using artificial-intelligence neural network models and evaluate the forecast performance of these novel models.
METHODS:
An innovative digital health solution, Patient-Responsive ED Intelligence on Consult Waiting Times (PREDICT), has been developed by adapting Deep Neural Network (DNN) models: convolutional neural networks (CONV) and long short-term memory networks (LSTM). DNN models were trained on the training dataset with an objective of minimising mean squared error (MSE). The forecast performance was evaluated using MSE, mean absolute prediction error (MAE) and percentage of underpredicted observations (PUO) on the test dataset. PREDICT performance was compared with the commonly used moving average (MA) method. PREDICT generated an aggregated ED waiting time forecasts in a time-series manner and provided a waiting time forecast with uncertainties to low acuity patients who arrived during a particular 30-minute time window. The comparison scenarios included one- and multiple-period-ahead forecasting.
2020 data from a large Australian tertiary hospital ED was used for analysis. Train dataset had 12893 records, validation dataset had 2532 records, and the test dataset included 705 records.
RESULTS:
Across all comparison scenarios, the accuracy performance of DNN ED waiting time forecasts was significantly higher than MA-based forecasts. For the one-period ahead forecasting, CONV outperformed the best MA model by over 35% with respect to MSE and reduced the number of patients with large underpredicted waiting times (> 30 minutes) by 77%. For the 12-period ahead direct-recursive forecasts (6 hours ahead), LSTM model was superior and outperformed the MA by 53% with respect to MSE.
CONCLUSIONS:
Our results suggest that flexible data-driven DNN models delivers more accurate and actionable waiting time forecasts than MA across one- and multiple-period ahead forecasts. Multiple-period-ahead forecasts have important practical benefits in improving ED waiting time management and responding to the ED demand more effectively.
Advancements in Digital Health Interventions: Evidence Syntheses, Value Assessment, and Global Perspectives
Digital Conference Pass
Discover the latest progress in digital health interventions. Explore evidence synthesis, collaborative value assessment, global perspectives, and cost-effectiveness across chronic disease management.
Moderator
Ramiro Gilardino, MD, MHS, MSc
MSD, Dubendorf, ZH, Switzerland
Dr. Ramiro Gilardino currently serves as Global HTA & Access Policy Leader at Merck & Co (known as MSD outside the US and Canada). With a 14-year background in health economics, outcomes research, and global health policy gained through experiences in pharmaceutical and medical device companies, as well as not-for-profit organizations, he actively engages in health technology assessment policies and patient engagement initiatives. His primary focus is on holistic value assessment, collaborating closely with decision-makers in established and emerging markets. His enduring commitment lies in driving global health innovation to ensure access to cutting-edge technologies while upholding the sustainability of healthcare systems
P33: Extending the PICOTS Framework for Evidence Syntheses of Digital Health Interventions: Results of a Delphi Consensus Study
1:45PM - 2:00PM
Zrubka Z 1 , Champion A2 , Holtorf AP3 , Di Bidino R4 , Earla JR5 , Boltyenkov A6 , Tabata-Kelly M7 , Asche C8 , Burrell A9 1 Óbuda University, Budapest, Hungary, 2 Healthcare Research Insights, Inc, Lake Forest, IL, USA, 3 Health Outcomes Strategies GmbH, Basel, Switzerland, 4 A.Gemelli Teaching Hospital, Roma, Italy, 5 Merck, Rahway, NJ, USA, 6 Siemens Healthcare Diagnostics Inc., Hellertown, PA, USA, 7 Brandeis University, Waltham, MA, USA, 8 University of Utah, Salt Lake City, UT, USA, 9 Anita Burrell Consulting LLC, Flemington, NJ, USA
OBJECTIVES: Previous research has shown that definitions of digital health-related terms are diverse and ambiguous. Therefore, we aimed to develop a minimum information framework for defining research questions for patient-facing digital health interventions that is sufficiently specific for evidence syntheses in health economics and outcomes research (HEOR).
METHODS: Building on a comprehensive review and qualitative content analysis of digital health- related definitions in systematic reviews, the ISPOR Digital Health Special Interest Group (DH-SIG) launched a Delphi survey study to reach consensus on items of a minimum information framework, following the CREDES guidelines. Altogether, 28 proposed subcategories were organized under the domains of PICOTS (“Population”, “Intervention”, “Comparator”, “Outcome”, “Timing” and “Setting”) extended with “Information”, “Communication”, and “Technology”. Volunteering members of the DH-SIG with at least moderate familiarity with digital health were invited to rate, comment and modify the proposed items in the first round of the Delphi survey, followed by two rounds of repeat ratings and feedback on the aggregated results. Consensus was predefined as ³70% “important” or “very important” ratings in the final round.
RESULTS: After screening 45 DH-SIG-members, 31 were eligible for the Delphi survey. The first, second and third rounds were completed partially or fully by 18, 11 and 10 respondents, respectively. The panel (18 first-round respondents) was diverse in terms of age, gender, geography, and professional background. Following panelists’ feedback, the domain “Context” and five subcategories were added in round two. In the third round, all 33 subcategories and nine of the ten domains reached consensus for inclusion in the minimum information set; with the “Information” domain excluded.
CONCLUSIONS: When defining PICOTS research questions on patient-facing digital health interventions in addition to the population, intervention, comparator, outcome, timing and setting, details related to communication, technology and context should be specified to aid evidence syntheses for HEOR purposes.
P35: How Perspective Affects the Cost-Effectiveness of Remote Patient Monitoring for Patients With Heart Failure in Different European Countries
2:15PM - 2:30PM
Mokri H 1 , Rutten-van Mölken M2 , van Baal P3 1 erasmus university, Den Haag, ZH, Netherlands, 2 Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, South Holland, Netherlands, 3 Department of Health Economics, Erasmus School of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
OBJECTIVES: Accelerated by the discussion of value frameworks, the “societal perspective ” continues to gain traction in health technology assessment (HTA) agencies. There is debate about which costs outside the healthcare sector to include, especially about “future costs of non-medical consumption ”. This study aims to assess the impact of adding the costs of informal care and costs of future (non)-medical consumption to the costs of healthcare and long-term care on the cost-effectiveness of remote patient management (RPM) for heart failure.
METHODS: We developed a Markov model with a lifetime horizon to assess the cost-effectiveness of non-invasive RPM compared with usual care (UC) from a societal and healthcare perspective in the Netherlands, the UK, and Germany. We included intervention costs, heart failure-related hospitalization and non-hospitalization costs, other medical costs unrelated to heart failure, informal care costs, and costs of non-medical consumption.
RESULTS: RPM increased life expectancy by 0.2 years. It reduced heart failure-related hospitalization costs, but total lifetime costs were higher than UC, in all three countries. From a healthcare perspective, the incremental cost-effectiveness ratios (ICERs) were €20,399, €17,479, and €19,294 in the Netherlands, the UK, and Germany, respectively. Expanding this perspective to include informal care costs, the ICERs increased to €22,490, €21,329, and €21,450 in the Netherlands, the UK, and Germany, respectively. Further expanding to a full societal perspective that includes non-medical consumption costs increased the ICERs to €35,970, €39,131, and €43,825 in the Netherlands, the UK, and Germany, respectively. Besides the costs of non-medical consumption, the ICER was sensitive to the effectiveness of RPM and utility values.
CONCLUSIONS: RPM is less cost-effective from a societal perspective than a healthcare perspective because of the costs of non-medical consumption in life years gained. However, given commonly used thresholds, RPM might still be considered cost-effective.
P36: Towards a Collaborative Value Assessment Framework for Provider-Facing Digital Health Technologies Used in Chronic Disease Management: Findings and Implications
2:30PM - 2:45PM
Main C 1 , Haig M2 , Kanavos P1 1 London School of Economics and Political Science, London, UK, 2 London School of Economics and Political Science, London, LON, UK
OBJECTIVES: Value frameworks play an essential role in evaluating health technologies, including wide-reaching digital health technologies (DHTs). However, assessing the value of provider-facing DHTs poses unique challenges due to their complexities, broad impacts and data-related risks. This study aims to develop a collaborative value assessment framework specifically for provider-facing DHTs used in chronic disease management and explore key stakeholder opinions on where value lies.
METHODS: Mixed methods comprising both primary and secondary evidence were utilized in this study. 34 indicators were proposed from an initial scoping review within six value domains: health inequalities, data rights and governance, technical and security characteristics, clinical characteristics, economic characteristics and user preferences. Subsequently, a three-round web-Delphi was conducted with 79 participants consisting of patients, HCPs, decision-makers, industry and academics (including policy experts). Three rounds were completed to update the framework and understand different stakeholders views on the indicators’ importance in the context of technology assessment.
RESULTS: The framework started at 45 indicators based on participant contributions in qualitative round one and delivered 16 stable indicators with consensus after rounds two and three. The highest-rated indicators included strong information governance standards, long-term cost-effectiveness, and data integrity. However, high rates of instability and dissensus were observed, particularly by clinicians and policy experts in the data rights and governance domain, emphasizing the complexity and novelty of assessing provider-facing DHTs. Stakeholders demonstrated a need for better understanding and discussions on the value, impact and infrastructure requirements of these technologies.
CONCLUSIONS: The collaborative value assessment framework provides valuable insights into the assessment of provider-facing DHTs, but further discussions and refinement are necessary to address the complexity and uncertainties associated with these technologies. Continued stakeholder involvement and knowledge exchange are crucial for advancing the understanding and evaluation of provider-facing DHTs within the healthcare ecosystem.
P34: Health Technology Assessments of Digital Public Health Interventions in the Southeast Asia - A Systematic Scoping Review
2:00PM - 2:15PM
Gudi N 1 , S EAR2 , Jahn B3 , Siebert U4 , Brand A5 1 Public Health Evidence South Asia, Prasanna School of Public Health, Manipal Academy of Higher Education, Manipal, Karnataka, India. Faculty of Health Medicine and Life Sciences (FHML), Maastricht University, Maastricht, Netherlands, 2 Public Health Evidence South Asia, Prasanna School of Public Health,Manipal Academy of Higher Education, Manipal, Karnataka, India, 3 UMIT TIROL - University for Health Sciences and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Hall i.T., 7, Austria, 4 UMIT TIROL - University for Health Sciences and Technology, Hall in Tirol, Austria. ONCOTYROL - Center for Personalized Cancer Medicine, Innsbruck, Austria. Harvard T.H. Chan School of Public Health and Harvard Medical School, Hall in Tirol, 7, Austria, 5 Faculty of Health Medicine and Life Sciences (FHML), Maastricht University, Maastricht, Netherlands. United Nations University – Maastricht Economic and Social Research Institute on Innovation and Technology (UNU-MERIT), Maastricht, Netherlands
OBJECTIVES: To map and analyze the Health Technology Assessments (HTA) of digital public health interventions in the WHO South East Asian region.
METHODS: A systematic scoping review was performed with searches conducted on electronic databases. We included HTAs of digital public health interventions and excluded narrative reviews, commentaries, and editorials. Screening at title-abstract and full-text stage was conducted by two reviewers, followed by data extraction. Dimensions of assessment were analyzed against the EUnetHTA Core model 3.0. Quality assessment of included articles were conducted using the JBI Checklist for Economic Evaluation and CHEERS 2022 checklist was used to assess the reporting quality.
RESULTS: We included 13 studies conducted across six countries for analysis. Of these, telemedicine and the m-health interventions were assessed in ten studies. Nine studies conducted cost-effectiveness analysis and 39% of the assessments were conducted from a societal perspective. Four studies utilized more than one perspective for assessment. Health problem and current use of technology (CUR), description and technical characteristics of technology (TEC), time horizon, clinical effectiveness (EFF), costs and economic evaluation (ECO) and organizational aspects (ORG) were assessed by all the studies. Legal aspects were least assessed among the core dimensions of HTA. Most studies were well conducted and well reported.
CONCLUSIONS: Although the reporting quality among the included studies were good, sufficiency of reporting needs attention. The limited set of HTAs of digital public health interventions highlights a stronger need for capacity building and context specific HTA frameworks to assess digital public health interventions. Evaluations should focus and report on aspects additional to cost, such as usability and feasibility aspects.
14:30 - 15:15
New Professionals Networking Reception
Join us for a chance to network with new and early professional HEOR colleagues from around the globe. Learn more about the ISPOR new professionals member group and how to get involved in a vibrant community and advance your career.
14:45 - 15:15
Coffee Break
Located in the Exhibit Hall
14:45 - 15:45
Member Group Meetings
Clinical Outcome Assessment Special Interest Group Open Meeting
The Clinical Outcome Assessment Special Interest Group invites you to join our Open Meeting where we will provide an overview of our ongoing activities and seek your input on upcoming project ideas and topics. We are currently developing an upcoming Key Project focusing on how clinical trials balance the evidence needs of regulatory and reimbursement bodies when assessing patient-reported outcomes. During the meeting, we will present the proposal for this project and discuss opportunities for members to join our Working Group. Your participation and insights are highly valued.
To view our SIG webpage, visit here . We look forward to sharing ideas with you, gaining your valuable feedback, and exploring ways for you to get involved!
New Professionals Networking Open Meeting
15:15 - 15:45
Exhibit Hall Theater
Healthcare System Benefits of Improving Patient Access to Early-Stage Cancer Treatments in Europe
Digital Conference Pass
Diagnosing and treating patients with cancer as early as possible can reduce the risk of recurrence and improve chances for a cure. Evidence also suggests that early action against cancer – including in the form of early-stage treatments – can have benefits for healthcare systems. While the clinical potential of medicines in early-stage cancers is increasingly acknowledged, challenges remain to patients’ access to these medicines across Europe. Based on a systematic analysis of these challenges, the session will discuss actionable solutions to address them from a policy and healthcare system perspective. A special focus will be placed on the economic value of early diagnosis, treatment, and well-functioning cancer patient pathways.
Sponsored by MSD
Sponsor
MSD
Speaker
Oriana Ciani, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Oriana Ciani is Associate Professor of Practice at SDA Bocconi in Milan. She holds a MSc in Biomedical Engineering from Politecnico di Milano and postgraduate degree in Healthcare Management from Bocconi University. She received her PhD from the University of Exeter with a thesis focusing on the evaluation of surrogate end points. She has been 2020 Fulbright Research Scholar at Yale School of Medicine and Yale School of Public Health. Oriana's research interests are centred on the use of evidence synthesis techniques to inform policy decisions, health technology assessment (HTA) and healthcare policies evaluation.
Ramiro Gilardino, MD, MHS, MSc
MSD, Dubendorf, ZH, Switzerland
Dr. Ramiro Gilardino currently serves as Global HTA & Access Policy Leader at Merck & Co (known as MSD outside the US and Canada). With a 14-year background in health economics, outcomes research, and global health policy gained through experiences in pharmaceutical and medical device companies, as well as not-for-profit organizations, he actively engages in health technology assessment policies and patient engagement initiatives. His primary focus is on holistic value assessment, collaborating closely with decision-makers in established and emerging markets. His enduring commitment lies in driving global health innovation to ensure access to cutting-edge technologies while upholding the sustainability of healthcare systems
15:15 - 16:15
Educational Symposia
Two concurrent sessions
Discussion Groups
How to Determine Unmet Needs for More Needs-Driven Product Development and Healthcare Decision-Making?
Moderator
Isabelle Huys, PharmD, PhD
Department of Clinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, VBR, Belgium
Prof. dr. Isabelle Huys is full professor at the University of Leuven (Belgium). She leads the research group on Regulatory Sciences at the Faculty of Pharmaceutical Sciences, embedding the Patient Evidence and Preference Center (PPRC). Her research focusses on market access of medicines and therapies, with particular attention to the inclusion of the voice of the patient in drug development, evaluation, market access and usage. In addition, ethical, legal and social issues (ELSI) are studied in an interdisciplinary context.
The "Population, Intervention, Comparator, and Outcome" Puzzle: Navigating Uncertainty in the Joint Clinical Assessment Era
Speaker
Wim Goettsch, PhD
National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands
Professor Wim Goettsch is a key contributor to European HTA. At Utrecht University he is a Professor of HTA of Pharmaceuticals. He has serves as a special HTA advisor to Zorginstituut Nederland and a former director of EUnetHTA JA3 Directorate.
ISPOR Forums
Implementing Novel Value Elements and Methods in Health Technology Assessment: The Case of Advanced Therapies
Objectives: Discuss whether conventional cost-utility analysis will continue to serve a meaningful purpose for the health technology assessment (HTA) of advanced therapies, or if novel value elements and methods will become the next evolution for the HTA of advanced therapies.Background: Conventional cost-utility analysis has been criticized for omitting many important elements of value, such as value of hope, loss of exclusivity, insurance value, real option value, equity, and severity. These novel elements of value may have particular relevance for the comprehensive value assessment of advanced therapies. Despite these value elements being omitted, conventional cost-utility analysis remains standard practice across many HTA bodies. Novel methodological advances such as generalized risk-adjusted cost-effectiveness (GRACE), distributional cost-effectiveness analysis (DCEA), and stacked cohort models for dynamic pricing may offer options to incorporate these additional elements of value quantitatively. At present, these methods are not traditionally implemented within HTA.Panel: Dr. Padula will introduce novel value elements (e.g., petals of the ISPOR value flower) and novel value methods (e.g., GRACE, DCEA, stacked cohort models) with an emphasis on their relevance for advanced therapies. Then the members of the panel representing England (Prof. Wailoo), Central/Eastern Europe (Ms. Khmelovska), and North America (Dr. Whittington) will first discuss how advanced therapies are traditionally assessed in their country followed by a debate on whether these elements and methods are currently feasible and ethical for implementation within the HTA for advanced therapies.
Moderators
William Padula, PhD, MSc, MS
University of Southern California, Los Angeles, CA, USA
William Padula, PhD is Assistant Professor of Pharmaceutical & Health Economics at the University of Southern California School of Pharmacy, and a Fellow in the Leonard D. Schaeffer Center for Health Policy & Economics. He is a Co-Founder & Principal at Stage Analytics. His research focuses on the theoretical foundations of medical cost-effectiveness analysis and applications of machine learning. He was the 2021 recipient of ISPOR’s Bernie O’Brien New Investigator Award, Co-Chair of the ISPOR Machine Learning Task Force, and is an Associate Editor for Value in Health.
Speakers
Marharyta Khmelovska, M.Ec.
State Expert Center of the Ministry of Health of Ukraine, Kyiv, Ukraine
Marharyta Khmelovska is an expert in the division of economic evaluation of health technologies at the HTA Department of the State Expert Centre of the Ministry of Health of Ukraine She is involved in the processes of conducting the state HTA in the part of the assessment of the pharmacoeconomic section of the dossier, taking part in activities aimed at implementing the tasks of the HTA road map in Ukraine and institutionalizing the HTA processes in Ukraine.
Allan Wailoo, MA, MSc, PhD
University of Sheffield, SHEFFIELD, United Kingdom
Allan Wailoo, PhD, is professor of Health Economics in the Sheffield Centre for Health and Related Research (ScHARR), University of Sheffield, UK where he has worked since 2000. He is Director of the NICE Decision Support Unit (DSU) and is Co-Director (with Mark Sculpher, York) of the UK Department of Health Policy Research Unit for Economic Evaluation.
Melanie Whittington, PhD, MS
Valusphere, Boston, MA, USA
Dr. Melanie Whittington is a Senior Fellow at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center and has a decade of experience in health economics and outcomes research. Most recently, she was the Director of Health Economics at the Institute for Clinical and Economic Review (ICER) where she led the cost-effectiveness analyses and budget impact analyses within ICER reviews. She was the 2022 recipient of the Bernie J. O’Brien New Investigator Award.
EU HTA Regulations (HTA-R) in Motion: Italy’s Path Forward
Italy has a highly regulated pricing, reimbursement, and access environment for Medicinal Products (MPs) and a rather de-regulated environment for Medical Devices (MDs). However, the current situation required significant changes to face the new challenges for the assessment and access of MPs and MDs, not only in Italy but also in all EU countries. The EU HTA Regulation (HTA-R) joint clinical assessment (JCA) requires health technology developers to submit a comprehensive clinical dossier to the EU Commission, from January 2025, for a selection of MP and MD, to be extended to larger groups of technologies in 2028 and 2030.
The Forum will address the interactions between the EMA process, the HTA-R and the local Italian pricing and reimbursement process for pharmaceuticals as well as the interactions between the HTA-R process and local Italian funding and access environment for devices.
Conti will present an overview of the Italian National HTA Program for medical devices 2023 2025 and how this program might affect the activities of medical device companies (10 min.); Berto will illustrate the challenges for drug manufacturers in dealing with EMA-HTAR-AIFA submissions as well as an update of the AIFA organization and governance (10 min.); Canonico and Cortesi will provide an Italian regulatory authority perspective (20 min.) and will facilitate the session and the interaction with the audience.
Audience participation will include identifying challenges in managing the EU HTA and Italian process interplay (20 min). The interactive element can be digitally enabled (app-based question & suggestion submission) and/or facilitated live by the moderators, as preferred by ISPOR and informed by venue and estimated attendee numbers.
This interactive and creative workshop will be valuable to industry HTA professionals, their colleagues in value & access strategy and management roles and to HTA agency staff.
Moderators
Pier Luigi Canonico, MD
University of Piemonte Orientale, Novara, Italy
Full Professor of Pharmacology since 1990 and Director of the Department of Drug Sciences at the University of Eastern Piedmont "Amedeo Avogadro" until November 2019.
He has been President of the Italian Society of Pharmacology (SIF), Member of the Unique Commission of Medicines of the Ministry of Health and Director of the Center of Excellence in Healthcare Management (CEIMS) the University of Eastern Piedmont "Amedeo Avogadro".
Member of the Board of Directors of the Italian Society of Neuropsychopharmacology. He is President of ISPOR Italy - Rome chapter from 2020 to 2023.
In addition, he is a member of numerous national and international Scientific Societies and author of more than 200 publications in international journals
Speakers
Patrizia Berto, PharmD,
AESARA Europe, Verona, VR, Italy
Patrizia Berto currently holds a position as Sr. Director Head of P&R at Pharmalex Italy and is a Director of the Italian (Rome) Chapter of ISPOR (2021-2023). Patrizia has spent 10 years in pharma at Glaxo and more than 25 years in consulting leading multiple Strategic Market Access, Pricing and Reimbursement and HEOR consulting initiatives for pipeline, launch and marketed products across a vast array of therapy areas, from rare diseases to oncology, haematology and many others. She holds a PharmD from Padova University and an MBA (CUOA - Veneto Region). In the years 2001-2013 she was a lecturer (contract professor) of pharmaco-economics at the University of Padova.
Francesco Marco Conti, LL.B.
Medtronic, Milan, MI, Italy
Dr. Francesco Marco Conti, born in Milan on 9 August 1961 and graduated in Business Administration at Bocconi University of Milan.
Government Affairs, Communication, Regulatory & Congress Director at Medtronic Italia S.p.A. and President of Fondazione Charta - Center for Health Associated Research and Technology Assessment
Paolo Cortesi, PhD
University of Milan-Bicocca, Monza, MB, Italy
Real-World Data Quality - Perspectives From Different Stakeholders
Global healthcare decision-makers require that real-world data (RWD) must be high quality and fit-for-purpose for real-world evidence (RWE) to be used in decision-making. While there is guidance available that discusses the parameters of high-quality data, such as completeness and accuracy, this guidance is often high level. This forum will focus on operationalizing data quality standards by discussing the following key topics in RWD quality:
A brief overview of the European Medicines Agency's (EMA) data quality framework
How to cope with data quality issues in RWE studies targeted for regulatory approval, post-approval safety, and/or health technology assessment (HTA) decision making
What information do regulators or HTA agencies need to feel confident about data quality?
How to collaborate with data providers to ensure data quality, trustworthiness, and transparency
This forum session will bring together analytical experts that have expertise in helping companies identify high-quality fit-for-purpose data sources, industry experts who are working to meet data quality standards in their RWE generation, and decision-makers who must judge the quality of RWD from submitted studies. Life sciences industry representatives across all segments as well as other interested stakeholders will benefit from attending this session.
Moderators
Doug Foster, MBA
Advanced Data Sciences, SAN FRANCISCO, CA, USA
Doug Foster is a Partner at Advanced Data Sciences (ADS), an advisory firm focused exclusively on the use of healthcare data for research and applications. He has spent over twenty-five years in healthcare innovation. Previously, Doug was Co-founder, Chief Executive Officer, and Chief Strategy Officer at Verana Health a real-world evidence company. Before this, Doug was Managing Director at Longitude Capital, where his focus was on early to late-stage venture capital investments in the life sciences industry.
Speakers
Megan O'Brien, PhD, MPH
Merck Sharp & Dohme International Service B.V., North Wales, PA, USA
Megan O’Brien, PhD, MPH, is is Associate VP and Head of the Global Center for Observational and Real World Evidence at Merck. In this role, Dr. O’Brien leads the Policy Evidence Research team. The focus of this team is to generate evidence to inform policy related discussions around access to pharmaceuticals, and to contribute to real-world evidence, HTA and value framework policies and standards development. In previous roles at Merck, Dr. O’Brien was responsible for the development of economic models for several pharmaceutical products and for value evidence strategies and RWE studies for vaccines. Dr. O’Brien received a PhD in health economics from the Johns Hopkins Bloomberg School of Public health and a Masters Degree in Public Health from Emory University.
Shaun Rowark, MSc
National Institute for Health and Care Excellence (NICE), Manchester, United Kingdom
I have worked in UK healthcare informatics for over a decade, starting out as a data analyst in hospital trusts to now being an associate director overseeing NICE’s access to healthcare data sources and developing its burgeoning data analysis function. I manage several programmes of work including automated reporting and computable guidance, helping to ensure that NICE’s products are suitable for a digital healthcare system.
Wendy Turenne, MS
Aetion, New York, NY, USA
Wendy Turenne is a healthcare analytics specialist with 20 years of experience building bridges between data, analytics, and business strategy. As SVP of Real-World Data and Operations, Wendy leads data strategy, data integration, and delivery operations for Aetion. Prior to joining Aetion, Wendy partnered with biopharmaceutical companies of all sizes on RWE and HEOR collaborations with Cardinal Health and DaVita. She previously held analytics and leadership positions across multiple settings, including government, population health, hospital, payer, and provider organizations. She holds a BS from the University of Virginia and an MS in biostatistics from the University of North Carolina, Chapel Hill.
Educational Symposia
An Eye for the Future: Does an Interdisciplinary View Help Improve Discounting Approaches for Curative Gene Therapies?
Digital Conference Pass
How much should we invest today to unlock health and well-being in the future? Current methodologies to answer this question require discounting future costs and benefits within an economic evaluation reflecting societal preferences for immediate impact. Yet, technological progress enables the generation of benefits ever longer into the future, amplifying the impact of most discounting practices on today's decision-making. Within health, this issue manifests itself notably in preventative interventions, including advanced therapy medicinal products (gene, cell and regenerative therapies) with long-term benefits. But other fields adjacent to health, most prominently climate change, experience the same issue: A long time lag between the costs of actions taken today and the related benefits in the future - be it health gained or tons of carbon saved - poses challenges to current investment decisions.
Sponsored by Office of Health Economics
Sponsor
Office of Health Economics
Moderators
Simon Brassel, MSc, Dipl.Ing
Office of Health Economics, London, United Kingdom
Simon is a Health Economist and Electrical Engineer and works as a Senior Principal Economist at the Office of Health Economics in London. He has over 10 years of professional experience in international healthcare markets covering the whole spectrum of health technologies, from pharmaceuticals to digital health technologies.
Before joining the OHE, Simon worked at the EMEA headquarters of Fresenius Medical Care, the world’s largest provider of products and services for patients suffering from chronic kidney disease. There, he held various roles within the field of therapy marketing and product management and in the field of Health Economics, Market Access and Policy Affairs.
Before Fresenius, Simon worked as a technology consultant at the VDI Technologiezentrum in Berlin. As part of a small team, he worked on the National Strategy Process on Innovations in Medical Technology, which three German Federal Ministries initiated. He also served as a grants officer and helped fund a range of collaborative research projects in the health-tech field with support from the German Federal Ministry of Research and Education.
Simon also holds an MSc in Health Policy (Health Economics) from the London School of Economics and Political Sciences (LSE), a Dipl.-Ing. in Electrical Engineering from the Technische Universität München (TUM), and a joint Diploma in General Management from the University of Rochester and the University of Bern.
Speakers
Avril Daly, BSc
Retina International, Zürich, Switzerland
Avril Daly has been President of EURORDIS-Rare Diseases Europe since November 2022. She was elected to the EURORDIS Board of Directors in 2009 and served as Vice-President of EURORDIS from 2012 to 2022.
She is CEO of Retina International, the global patient-led umbrella group dedicated to the promotion of research into inherited and age-related retinal dystrophies. Retina International acts as a voice for patients living with retinal disease on policy issues that affect them and fosters cooperation among the medical and scientific communities with a view to expediting the development of and access to treatments for unmet needs.
Avril previously held the role of CEO at the Irish charity Fighting Blindness where she was responsible for the implementation of the organisation’s strategic development plan.
She a Board Member of Rare Diseases Ireland and in 2011 was appointed by the Minister of Health to the steering committee working towards the development of the Irish National Plan for Rare Diseases, published in 2014. Avril sat on the oversight committee reviewing the implementation of that plan and on the working group of the Clinical Programme for Rare Diseases at the Irish Health Service Executive (HSE).
She was diagnosed with the rare retinal condition Retinitis Pigmentosa in 1998.
Mohamed El Alili, PhD
National Health Care Institute, Diemen, Netherlands
Mohamed El Alili works as a senior pharmaco-economic advisor for the National Health Care Institute (Zorginstituut Nederland). His main task involves the assessment of cost-effectiveness of new pharmaceuticals. Other projects he's working on are the update of the pharmaco-economic guideline and the implementation of R within the National Health Care Institute. He has a PhD in statistical methods for trial-based economic evaluations obtained from the Vrije Universiteit Amsterdam. He is also affilitiated with the Vrije Universiteit Amsterdam where he works as a postdoctoral researcher at the Department of Health Sciences.
Grace Hampson, MSc
Office of Health Economics, London, United Kingdom
Grace is an economist with expertise in the methods and processes of value assessment, reimbursement and healthcare decision making. Her current research interests include interactions between health technology appraisal and novel payment mechanisms, particularly in relation to innovative technologies such as cell and gene therapies and new drugs to tackle Antimicrobial Resistance. Grace leads OHE’s Policy Organisation and Incentives in Health Systems research team.
Nicola Clair Trevor, MSc
Janssen, High Wycombe, United Kingdom
Nicola has over 13 years experience in health economics, market access and reimbursement. Her experience spans pharmaceuticals (local operating company and regional roles), health technology assessment bodies and consultancy. Entering health economics from a predominantly technical route, Nicola has rapidly developed passion and aptitude for innovative strategic approaches. Nicola is committed to changing the HTA and pricing environment to better deliver for patients, the NHS and the life science industry.
Time Delay – A Meaningful Outcome of Disease Modification in Alzheimer’s Disease
Digital Conference Pass
Alzheimer’s disease is a progressive neurologic disorder, with symptoms gradually developing over multiple years. Symptoms often begin with mild cognitive impairment and, over multiple years, gradually lead to severe loss of cognitive function.
The slow progressing nature of the disease makes it challenging to demonstrate benefit of potential therapies. Specifically, both manufacturers and HTA bodies have found it difficult to assess and demonstrate the efficacy of potentially disease modifying therapies that aim to slow progression of neurodegenerative diseases such as Alzheimer’s.
However, a novel assessment method, focused on measuring the slowing of disease and resultant prolonging of time to disease progression has been proposed, leading to the question: how can this novel outcome measure support value demonstration and assessment?
This session will include perspectives from multiple stakeholders on:
Why prolonging the time until progression is important and has been proven difficult to demonstrate to date
How the novel measure can help in overcoming historical barriers in effect demonstration and assessment
Its potential application to health economic models and HTA assessment
Sponsored by Novo Nordisk
Sponsor
Novo Nordisk
Moderators
Max Schlueter, MSc
IQVIA, London, United Kingdom
Max Schlueter is a Principal for Real World Methods and Evidence Generation at IQVIA. He leads diverse Health Economics & Outcomes Research (HEOR) projects and real world research studies for clients from the public sector and the life sciences industry. He holds an MSc in Health Policy, Planning and Financing from the London School of Economics and the London School of Hygiene and Tropical Medicine.
Speakers
Julie Hviid Hahn-Pedersen, MSc
Novo Nordisk A/S, Copenhagen, Denmark
Julie Hahn-Pedersen is an Associate Director, Global Market Access, Novo Nordisk, where Julie is responsible for developing evidence strategies for Novo Nordisk medicines. Julie has 10 years of experience within the pharmaceutical industry, working in different therapeutic areas, in different companies across the value chain. Julie has a Bachelor degree in Medicine, a Master degree in Biomedicine and a Master degree in Health Economics.
Linus Jönsson, MD, PhD
Karolinska Institutet, Stockholm, Sweden
Linus Jönsson is professor of Health Economics at the department of Neurobiology, Care Sciences and Society, Karolinska Institute, Stockholm, Sweden. His research interests include economic evaluation of new diagnostics and therapies for Alzheimer's disease and other neurodegenerative disorders. Dr Jönsson is the author of over 110 original publications, reviews and book chapters and serves as scientific advisor to the Swedish government and National Board on Health and Welfare, and he is a co-organizer of the International Pharmacoeconomic Collaboration on Alzheimer’s Disease (IPECAD).
Birgitta Martensson, MBA
Person Living with AD and Former CEO, Alzheimer Schweiz Suisse Svizzera, Épalinges, Switzerland
Born in Sweden 1950, studied in Switzerland. Worked in insurance company, marketing company and finally as CEO of Swiss Alzheimer Association until 2001.
Anja Schiel, PhD
Norwegian Medicines Agency (NoMA), Oslo, 02, Norway
Anja Schiel works as Special Adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects at the Norwegian Medicines Agency. She has been Chair of EMA’s Biostatistics Working Party 2017 – 2019 and from 2019 – 2022 she was Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member at the SAWP and is member of the new Methodology Working Party (MWP) recently established at EMA.
She has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21 with a particular focus on parallel EMA-HTA scientific advices.
15:30 - 16:15
Poster Tours
Student Research Spotlight
Posters featured in this tour:
PT43: Cost-Effectiveness Analysis of Prostate-Specific Antigen (PSA)-Based Risk-Adapted Screening in Germany: An Early Economic Evaluation Study
PT44: Cost-Effectiveness of Olaparib Vs. Rucaparib for Patients with Metastatic Castration-Resistant Prostate Cancer – the Canadian Perspective
PT45: Disease Relapse and Adverse Events During the Combination Use of Clozapine and Long-Acting Injectable Antipsychotics and During the Clozapine Monotherapy
PT46: Does Public Research Investment on Emerging Infectious Diseases Correspond to Disease Burden in China? A Cross-Sectional Study from 2009 to 2019
PT47: High-Volume Hospitals Experience Fewer Postoperative Complications After Neonatal Surgery: Analyses of the National Clinical Database Pediatric Surgical Registry in Japan
Sponsor
Moderator
Isabelle Borget, PharmD, PhD
Master2 Market-Access and Economic Evaluation, Orsay, France
Professor in Health economics and market access
Guvenc Kockaya, MD, MSc, PhD
ECONiX Research, Samsun, 55, Turkey
Dr. Guvenc Kockaya is a medical doctor and health economist. He has a career of more than 15 years in market access & health economics with government, academia, and private industry experience. He is the editor of the books titled as “Pharmaceutical Market Access in Emerging Markets” and “Pharmaceutical Market Access in Developed Markets” which are still in the Top 100 Best Sellers (International) in Amazon under “Pharmaceutical & Biotechnology Industry (Kindle Store)”. He is the founder of ECONiX which is an internal company that gives tailor-made consultancy services including but not limited to market access, health economics & outcome research, medical affairs, and business development for government and academic institutes, pharmaceuticals, medical devices, and healthcare services companies in Eastern Europe, Middle East, North Africa, and Western Asia countries with offices in Estonia, Tunisia, and Turkey.
Rare Diseases Poster Tour
Posters featured in this tour:
PT37: A Systematic Review of Modelling Approaches in Economic Evaluations of Treatments for Inherited Bleeding Disorders
PT38: Budget Impact Analysis of rFVIII Glycopegylated-Exei for the Treatment of Haemophilia A in Colombia
PT40: Epidemiology and Burden of Illness of Lupus in Children by Form of Lupus: A Retrospective Longitudinal National Hospital Claims Study in France
PT41: Real-World Evidence in Rare Diseases in France: Treatment Patterns, Healthcare Resource Use, and Related Costs in Von Willebrand Disease (FORvWARD Study)
PT42: The Impact of Spinal Muscular Atrophy Type 2 on Caregivers in Argentina: Results of a Global Survey
Sponsor
Moderator
Mats Rosenlund, PhD
Daiichi Sankyo, Munich, AB, Germany
15:30 - 18:30
Poster Session 4
Live
15:45 - 16:15
Exhibit Hall Theater
Responding Faster to Market and Safety Signals: The Power of Real-Time EHR Data for Evidence Generation
Digital Conference Pass
The ability to respond rapidly to market and safety signals is critical for protecting patient safety, maintaining regulatory compliance, driving innovation, and achieving commercial success. Complete real-world data from EHRs can enable biopharma and medical device manufacturers to be proactive and agile in addressing these signals—especially when the data are available in near-real time and provide deep clinical context. In this presentation, we’ll highlight examples of how Truveta Data has been used to monitor product safety and effectiveness, understand complex and emerging conditions, monitor off-label prescribing trends, and more.
Sponsored by Truveta
Sponsor
Truveta
Speaker
Ryan Ahern, MD
Truveta, Bellevue, WA, USA
Dr. Ryan Ahern is Chief Medical Officer and co-founnder of Truveta and has more than a decade of experience in healthcare data and clinical research. At Truveta, Ryan leads the Life Science Partner team which focuses on developing new partnerships and ensuring those partners are successful in their research. He is currently a Clinical Assistant Professor of Medicine at the University of Washington School of Medicine and practices at Harborview Medical Center. Previously, he trained at the Massachusetts General Hospital and served as a Clinical Assistant Professor at Weill Cornell Medical Center and New York Presbyterian; attending physician at Massachusetts General Hospital; and instructor at Harvard Medical School.
Katia Hannah, PhD
Dexcom, San Diego, CA, USA
Dr. Hannah has extensive experience conducting real-world evidence studies and HEOR research. Her role as a HEOR Research Specialist at Dexcom involves developing and implementing HEOR analytic strategies and executing value driven real-world data analytics utilizing multiple real-world data sources. Prior to joining Dexcom, she received her PhD in Pharmaceutical Outcomes Research and Master of Public Health from the University of Colorado. Dr. Hannah has presented her research at international conferences and has authored or co-authored 10 published scientific papers and abstracts.
Fast Facts
New Good Practices Recommendations for Publications
Level: Introductory
Discussion Leader
Eline Hanekamp, PhD, CMPP
Excerpta Medica, Amsterdam, Netherlands
Eline is Program Director at Excerpta Medica. For 13+ years, she has provided medical and HEOR publication planning services for large pharmaceutical and small biotech companies, working on blockbuster drugs, niche therapies and medical devices. Her teams provide strategic and practical support in publication planning, content development, and education, targeting global and regional medical and HEOR audiences. Eline serves as European Trustee on the ISMPP Board of Trustees, is a member of the GPP Steering Committee and co-author on GPP2022.
16:00 - 16:45
Network, Collaborate, and Get Involved in ISPOR's Global Groups
Are you interested in getting involved in the development of HEOR globally in support of ISPOR's mission? Join us to learn more about Global Engagement Groups and how you can be part of the global groups in North America, Latin America, Middle East, Africa, Europe, and Asia Pacific.
16:15 - 17:15
Member Group Meetings
Statistical Methods in HEOR Special Interest Group Open Meeting
Want to see what is up and coming in the field of statistical methods in HEOR? Come by the Statistical Methods in HEOR Special Interest Group open meeting on
Tuesday, November 14 at 16:15 to join in the conversation on future projects and existing work related to external control arms!
The group is also excited announce new additions to the SIG leadership team during this meeting, discuss the recently published article "Handling Missing Data in Health Economics and Outcomes Research (HEOR): A Systematic Review and Practical Recommendations,” as well as brainstorm future key project topics with through conversation with current SIG leadership. We hope that you will join us for an exciting and engaging hour while we share this past year's accomplishments and look to the future!
Looking forward to seeing you there!
16:15 - 17:45
ISPOR Spain Chapter Meeting
This meeting is open to all who are interested in learning more about and getting involved in the activities of ISPOR Spain Chapter.
17:00 - 18:00
Breakouts: IP, WS, & OBS
Time to Implement Multi-Level Network Meta-Regression (ML-NMR) Rather Than Matching-Adjusted Indirect Comparisons
Digital Conference Pass
Level: Intermediate
PURPOSE: Multi-level network meta-regression (ML-NMR) extends the standard network meta-analysis framework to leverage individual patient data and aggregate data when comparing multiple treatments while adjusting for differences in populations between trials. Unlike previous population adjustment approaches, ML-NMR is applicable in networks of any size, avoids aggregation bias and issues with non-collapsible effect measures, and crucially for decision-making produces estimates in any target population.
DESCRIPTION: Workshop attendees will obtain a working knowledge of the ML-NMR method, its advantages, and considerations for implementation. Dr. Jansen will chair the session and introduce ML-NMR in the context of the challenges with existing methods (10 min.). Dr. Phillippo will explain the statistical methods for ML-NMR, highlight advantages relative to existing methods, and provide an overview of how to implement the method using the multinma R package in terms of the syntax and features (15 min.). Ms. Cope will illustrate how these methods can be applied in a case study regarding the comparative efficacy of alternative interventions for triple-class exposed relapsed refractory multiple myeloma. This will include audience participation regarding selection of covariates, alternative time-to-event models, conditional vs. marginal estimates, and target populations for prediction (15 min). Mr. Klijn will describe lessons learned and recommendations for implementation of ML-NMR (15 min). Questions from the audience will be addressed (5 min) and this interactive workshop will be valuable to researchers and industry analysts interested in comparative efficacy research for health technology assessments.
Discussion Leaders
Jeroen Jansen, PhD
University of California – San Francisco, San Francisco, CA, USA
Jeroen P Jansen PhD is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics.
He is an Associate Professor in the Department of Clinical Pharmacy in the School of Pharmacy at the University of California, San Francisco and Chief Scientist − Health Economics & Outcomes Research at the Precision Medicine Group.
For the past 15 years, Dr. Jansen has worked on research to understand the clinical and economic value of healthcare interventions. His research has frequently been conducted in the context of health technology assessment (HTA) with a focus on comparative effectiveness and cost-effectiveness. Prompted by the challenges encountered in applied research projects, he has performed methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. He has promoted a more transparent and credible approach to model-based health economic evaluations and led the development of open-source simulation models to illustrate its feasibility. Furthermore, Dr. Jansen has been involved in the ongoing development of an R software package to develop simulation models for health economic evaluations.
His current research interests are the clinical and economic value of precision medicine, incorporating health disparities in health economic modeling studies, and statistical methods for evidence synthesis.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Discussants
Shannon Cope, MSc
PRECISIONheor, Vancouver, BC, Canada
Shannon Cope is a Senior Vice President with PRECISIONheor with fifteen years of experience consulting for biopharmaceutical industry. Shannon leads evidence synthesis projects involving (network) meta-analyses, population-adjusted indirect comparisons, multi-level network meta-regressions, surrogacy, and economic models. Her publications relate to application and extension of evidence synthesis methods related to (network) meta-analysis, the feasibility assessment process, time-to-event outcomes, and the integration of individual patient data from clinical trials, real-world evidence, and expert opinion. She has an MSc in Health Administration from the University of Toronto, Canada.
Sven Klijn, MSc
Bristol-Myers Squibb, Utrecht, ZH, Netherlands
Sven Klijn is director at Bristol Myers Squibb in the HEOR Economic & Predictive Modeling group, where he leads the innovative modeling agenda in hematology. In addition, Sven has an active role in providing modeling education and masterclasses at international congresses. He has widely published on innovative methods, especially in the field of survival extrapolation. Sven has a training in public health and health economics and previously had various roles in CROs related to health-economic modeling.
David Phillippo, PhD, MSc
University of Bristol, Bristol, BST, United Kingdom
David's research focuses on methods for evidence synthesis, Bayesian network meta-analysis (NMA), population adjustment methods for indirect comparisons, and assessing the impact of bias in clinical guidelines and decision making. He is the lead author of Technical Support Document 18 published by the NICE Decision Support Unit on population-adjusted indirect comparisons, providing guidance on the use of this new class of methods in NICE Technology Appraisals. He is a member of the NIHR Bristol Technology Assessment Group, a network member of the NICE Decision Support Unit, and he supports the development of NICE Clinical Guidelines through his involvement with the NICE Technical Support Unit based in Bristol. He is the author and maintainer of several freely-available R packages, including nmathresh for assessing sensitivity to biased evidence in NMA using threshold analysis, and multinma for performing NMA and multilevel network meta-regression with individual and aggregate data.
Real-World Impact of Real-World Evidence Guidelines: Monitoring the Influence of International Regulator and HTA Guidance With Key Case Studies
Digital Conference Pass
Level: Intermediate
PURPOSE: To provide a high-level overview of real-world evidence guidance from regulators and HTA agencies, exploring indicators of their influence and opportunities for greater alignment.
DESCRIPTION: In recent years, regulators and HTA bodies have signalled their intent to make greater and better use of routinely collected data and real-world evidence (RWE) to support their decision making processes.
This movement has resulted in the publication of several pieces of guidance from regulators including multiple FDA guidance on assessing electronic health records and medical claims data, and registries for decision making and multiple EMA guidance on data quality and methodological standards in pharmacoepidemiology. Meanwhile, HTA agencies have also produced frameworks including NICE’s RWE framework, CADTH’s framework for reporting standards for RWE studies, and the REALISE framework which supports the use of real-world data and RWE to support drug reimbursement decision making in Asia. However, to what extent can we currently see the influence of these publications – in terms of their application in evidence submissions, and the expansion of different RWE use cases to support decision making? Are they sufficiently aligned to support joint scientific advice both laterally (regulator to regulator and HTA to HTA) and vertically (from regulator to reimbursement)? The panel will start with an overview of published and drafted frameworks to guide the use of real-world data and RWE for regulatory and reimbursement decisions, with similarities and differences highlighted. Following this, regulatory and HTA representatives will present exemplary case studies showing their guidance in action. Next the perspective of an industry representative applying a guidance in their evidence submission will be provided. The session will end with interactive polls and a discussion of the main challenges impacting the application of these guidance, key areas missing, and the potential for greater alignment between different HTA bodies, and with regulators.
Discussion Leaders
Stephen Duffield, PhD, MD
National Institute for Health and Care Excellence, Liverpool, United Kingdom
Stephen is an Associate Director of Real-world methods at NICE. His role involves the continuing development of NICE’s real-world evidence (RWE) framework, collaboration on RWE demonstration projects, and helping to transform NICE’s use of real-world data across guidance products. He is also involved with upskilling individuals within and externally to the organisation, contributing to training workshops and technical forums. Stephen has a degree in medicine and a PhD in public health. Previously, he worked as a clinical doctor and a guideline developer in NICE Centre for Guidelines
Discussants
Juan Jose Abellan, PhD
European Medicines Agency, Amsterdam, Netherlands
Juan Jose Abellan is a mathematician and statistician by training. He has worked as a statistician in various roles in Public Offices, Academia and the Pharmaceutical Industry in Spain, Germany and the UK. He has worked in several scientific disciplines such as Epidemiology, Metagenomics and Clinical Drug Development, and he has made a number of contributions in those fields in peer-review scientific journals. He is currently working for the European Medicines Agency (EMA) based in the Netherlands and is part of the EMA team developing the Data Analysis and Real World Interrogation Network (DARWIN EU) for the generation of real-world evidence to support regulatory decision making around the utilisation, effectiveness and safety of medicines.
Tanja Podkonjak, MBA MSc
Takeda Pharmaceuticals International AG, Zurich, ZH, Switzerland
Hwee-Lin Wee, PhD
National University of Singapore, Singapore, Singapore
A/P WEE Hwee Lin is Associate Professor at the Saw Swee Hock School of Public Health at the National University of Singapore. She is a member of the International Society for Pharmacoeconomics and Outcomes Research Health Sciences Policy Council, member of the International Editorial Advisory Board for Journal of Patient Reported Outcomes, and member of the National Advisory Committee on Cancer in Singapore. A/P Wee’s current work focuses on the use of real world evidence in health technology assessments.
External Control Arm Planning for Rare Diseases
Digital Conference Pass
Level: Intermediate
PURPOSE: External control arms (ECAs) created from real-world secondary data sources are increasingly used for rare diseases to support applications to regulatory and health-technology assessment (HTA) agencies. The acceptance of ECA evidence by these agencies will depend heavily on the availability of a well-characterized external cohort and an appropriate justification of the sources and methods used.
In this workshop we will guide the audience through planning considerations for the creation of ECAs for regulatory and reimbursement purposes. Speakers will use real-time polling upfront to get to know the audience (5 min.), will ask each other questions during the presentations and will conclude with an interactive audience discussion (15 min.) to understand the audience’s opinions and experiences.
DESCRIPTION: Carla Vossen will provide a high-level overview of available ECA guidance, examples of ECAs used in regulatory and HTA submissions for rare diseases and the impact of EU Joint Clinical Assessment (10 min.). Raymond Huml will then present how data from different sources can be used for creating ECAs for clinical trials for novel muscular dystrophy treatments, what challenges may be faced and what solutions can be implemented (10 min.). Tracy Mayne will continue the discussion by presenting his recent experience with ECAs created from real-world claims and patient registry data to support regulatory submissions for primary biliary cholangitis (10 min.). Specific topics that Tracy Mayne will address are differences between EMA and FDA requirements (e.g., endpoint definitions, data sources, etc.) and key lessons learned. Lastly, Dawn Lee will discuss how to plan for implementing ECAs created from secondary real-world data sources in economic models for rare cancer indications, how to assess data quality and how to increase the chances of a successful reimbursement application (10 min.). This interactive workshop will be of interest to RWE users planning for ECAs and HTA practitioners.
Discussion Leaders
Raymond A Huml, MSc, DVM
Syneos Health, Morrisville, NC, USA
Raymond A. Huml, MS, DVM, RAC is Vice President of Medical and Scientific Strategy, Head of the Rare Disease Consortium and co-founder and Executive Sponsor of the Persons with Disabilities Employee Resource Group at Syneos Health.
Dr. Huml has over 35 years of experience in the healthcare and biopharmaceutical industries and a career spanning over 30 years in the Contract Research Organization (CRO) industry. He previously served as Head of Global Biosimilars Center of Excellence (for a Fortune 500 company) and as Head of Global Due Diligence where he identified risks associated with global, product-based investments (including rare disease investments), which resulted in almost $3.0b in capital committed to partnerships of all sizes.
Dr. Huml has authored or co-authored over eighty articles, over a dozen book chapters and four books on due diligence, competitive intelligence, muscular dystrophy, and rare disease drug development, respectively.
He was awarded North Carolina Veterinary Medical Association's Young Veterinarian of the Year Award and Regulatory Affairs Professionals Society’s New Professional Award. He has also received numerous CRO awards including the Distinguished Performance Award, Clinical Development Services President's Award, and the Chairman's Award.
Dr. Huml is a Returned Peace Corps Volunteer having served in Ghana, West Africa and sat for two terms on North Carolina State University’s Board of Visitors. He holds an MS in Biology from East Stroudsburg University and his DVM from North Carolina State University’s College of Veterinary Medicine and has earned the RAC (US) Certification.
Current or historical membership includes:
• National Organization of Rare Disorders (NORD) – Corporate Council Member
• US Center for Disease Control’s MDStarNet council representing persons afflicted with muscular dystrophy
• Founder and Member of the North Carolina Chapter of the Facioscapulohumeral Muscular Dystrophy (FSHD) Society
o Member: FSHD Society’s Health Economic Task Force
• Drug Information Association’s (DIA) Innovative Designs Scientific Working Group (IDSWG); Master Protocol Initiative: Patient Engagement Subgroup
• Berkeley’s Forum for Collaborative Research’s Bayesian Methods in Rare Diseases working group, UC Berkeley, School of Public Health
• Association for Regenerative Medicine (ARM) Foundation
o Member: State Rare Disease Education Initiative (STRiDE)
• Parent Project Muscular Dystrophy’s (PPMD) Community-led FDA Duchenne muscular dystrophy group to support an FDA draft guidance to the FDA
Discussants
Dawn Lee, MMath, MSc
University of Exeter, Exeter, United Kingdom
Dawn is an Associate Professor of Health Economics and Health Policy, working with the Peninsula Technology Assessment Group (PenTAG), one of 11 research units in the UK providing expert advice on the clinical and cost-effectiveness of new drugs to the National Institute of Health and Care Excellence (NICE). She is also an existing member of the NICE interventional procedures appraisal committee.
Dawn joined PenTAG / Exeter University in Sept 2022 following over 15 years in economic consultancy where she worked most recent as the Chief Scientific Officer for a medium sized health economics consultancy (Lumanity; formerly BresMed). She is a health economic modeler who has conducted over 50 UK Health Technology Assessment submissions and worked in over 30 countries globally, a member of NICE’s interventional procedures advisory committee (IPAC) and the R for HTA group. Dawn’s key project achievements include representing manufacturers at ~30 HTA Committee meetings, working on the first ever immune-oncology submission to NICE (TA268), first ever PD-1 submission to NICE (TA384) and a considerable number following this and the first ITC accepted by G-BA.
Dawn’s main research interests are oncology modelling; particularly immune-oncology and therefore flexible survival modelling and the incorporation of external data within extrapolations, structured expert elicitation and improving diagnostic pathways.
Dawn is currently working on NICE's pathways pilot.
Tracy J. Mayne, PhD
Intercept Pharmaceuticals, Morristown, NJ, USA
Tracy Mayne, PhD, is Vice President Global Medical Affairs at Intercept Pharmaceuticals.
Dr. Mayne has over 20 years of experience in consultancy and biopharmaceutical industries in medical affairs, epidemiology and health economics and outcomes research. His core focus and strengths lie in creating and executing successful medical affairs vision, strategic and tactical plans, successfully launching new drugs and maximize global price, access and reimbursement, building highly skilled teams to meet revenue and operating margin targets, effectively integrating into and working with development, commercial and government affairs teams. Key areas of experience are: rare disease, nephrology, hepatology, hematology, oncology, cardiovascular disease and pain.
Professional Affiliations
National Pharmaceutical Council: Board member, 2015-2018
AASLD
EASL
ACG
AMCP
ISPOR
Carla Vossen, PhD
Syneos Health, Amsterdam, Netherlands
Carla Vossen, PhD, is a Senior Director Epidemiology, RWE at Syneos Health.
Dr. Vossen has over 20 years of experience in epidemiological research as a clinical epidemiologist in academia and as an HEOR/epi/HTA consultant for the pharmaceutical industry. She has experience in observational studies from start to end, including design of externally controlled studies. Therapeutic and methodological topics of interest include innovative observational study designs, rare disease, oncology and genetics/genomics.
Training Patients in Europe for Effective Contribution to Health Technology Assessment
Digital Conference Pass
Level: Intermediate
Education and training preparing patients to effectively contribute to HTA processes need to be easily and widely available in order to support the implementation of the new European Health Technology Assessment (HTA) Regulation ((EU) 2021/2282) with useful and relevant input from patients and patient representatives. The European Patients’ Academy on Therapeutic Innovation(EUPATI) is experienced with patient education and aims to provide accessible, innovative and inclusive education empowering patients and patient representatives to effectively engage and partner with all other stakeholders. EUPATI is now joining forces with various partners, EUPATI National Platforms and trained expert patients to provide the training required for contributing to HTA.
This forum will focus on the type of information and training needs that patients may have to be able to contribute effectively to HTA processes as trusted partners in the context of the new HTA Regulation.
Attendees will be able to contribute to shaping a vision for patient education that enables meaningful patient involvement in the context of the new HTA Regulation. Maria Dutarte (EUPATI) will chair the session. Dr Anke-Peggy Holtorf (HTAi) will introduce the broader context of patient involvement in HTA, how this is adopted in the new HTA Regulation, highlighting challenges and opportunities. Maria Dutarte will present the EUPATI HTA4Patients project and the multi-stakeholder approach taken to design an ad-hoc patient education programme in the field of HTA. Lotte Klim (EUPATI Fellow) will make a case as to why it is important to empower patients in HTA and support them becoming trusted partners. The target audience including patients and patient representatives, researchers from both academia and industry, HTA body representatives with an interest in impactful patient involvement will be able to actively contribute by identifying relevant aspects to include for the patient training material (25 minutes).
Moderators
Maria Dutarte, MS
EUPATI, Utrecht, Netherlands
Discussion Leaders
Anke-Peggy Holtorf, PhD, MBA
PCIG @ HTAi.org, Basel, Switzerland
Anke-Peggy is the founder of the Health Outcomes Strategies GmbH consulting agency (www.health-os.com) with focus on value of healthcare products and related policy decisions for the in- and off-patent sector, reimbursement strategies, outcomes research and health economics, health technology assessment processes, payer interactions, product / device / service synergies.
Anke-Peggy acted as evaluator at the EU-commission on multiple occasions and is faculty member at the University of Utah College of Pharmacy where she reads in International Health Policy. She is engaged in workgroups for Precision Medicines Value (ISPOR), for Digital Health (ISPOR) and for Patient and Citizen Involvement (PCI) in HTA (as project coordinator for PCIG at HTAi) and leads projects on PCI in Low-and Middle-Income Countries and on PCI processes in HTA.
Lotte Klim, EUPATI Fellow
EUPATI, Copenhagen, 84, Denmark
The Novel Toolbox Quartet for Using Real-World-Data in Health Technology Assessment: Causal Diagrams, Target Trial Emulation, G-Methods, and Causal Modeling
Digital Conference Pass
Level: Introductory
PURPOSE: The objectives of this educational, practice-oriented and interactive lecture are to (1) introduce the modern toolbox needed to derive answers from real-world evidence (RWE) for decision making authorities, (2) provide applied case examples, and (3) discuss current challenges and solutions. DESCRIPTION: One of the most important tasks of health sciences researchers is to support decision makers with causal interpretations derived from real-world data (RWD). This lecture has four parts. First (30 min), an educational introduction of the four causal toolbox elements: Causal Diagrams, Target Trial Emulation, g-Methods, and Causal Modeling. Causal diagrams are used to assess the causal interpretability of intervention effects. Target trial emulations are used to avoid self-inflicted biases (e.g., immortal time bias) and to transport study results to target populations of interest. G-Methods are used to control for time-varying confounders (i.e., confounders simultaneously affected by treatment), which are present in most RWD concerning chronic disease treatments. Causal modeling includes structuring decision models causally, and carefully selecting causal model input parameters. This lecture part includes real-time polling for knowledge self-check. Second (15 min), real case examples from oncology, cardiovascular and infectious disease are used to demonstrate challenges and applied solutions, and to understand biases that arise when missing one of the toolbox elements. Examples include the application of the novel counterfactual “data cloning” approach. The lecturer will compare HTA bodies from different countries regarding the use of causal RWE approaches. Third (15 min), a structured discussion/survey with the audience using a unified terminology along the four toolbox elements will provide participants the opportunity to translate the lecture content to their own problems and to contribute with their own experiences. This lecture may guide researchers on RWE generation, RWD analysis, and causal modeling supporting approval and HTA decisions and provide didactical skills to educate their peers.
Speaker
Uwe Siebert, MD, MPH, MSc, ScD
UMIT TIROL - University for Health Sciences and Technology, Hall in Tirol, Austria. ONCOTYROL - Center for Personalized Cancer Medicine, Innsbruck, Austria. Harvard T.H. Chan School of Public Health and Harvard Medical School, Hall in Tirol, 7, Austria
Uwe Siebert, MD, MPH, MSc, ScD, is a Professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), Chair of the Department of Public Health, Health Services Research and HTA at UMIT TIROL - University for Health Sciences and Technology in Austria and Director of the Division for HTA in the ONCOTYROL–Center for Personalized Cancer Medicine in Austria. He is also Adjunct Professor of Epidemiology and Health Policy & Management at the Harvard T.H. Chan School of Public Health and Affiliated Researcher in the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston.
After medical school, he worked for several years as a physician in international public health projects in West Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in Decision Sciences at the Harvard School of Public Health.
His research interests include applying real-world evidence-based quantitative, causal and translational methods from public health, epidemiology, artificial intelligence, comparative effectiveness research, health services and outcomes research, economic evaluation, modeling, and health data a d decision science in the framework of health care policy advice and HTA as well as in the clinical context of routine health care, clinical guideline development, public health policies and patient guidance. His research focuses on cancer, infectious disease, cardiovascular disease, neurological disorders, and others.
He has been leading projects/work packages in several EU FP7, H2020 and Horizon Europe projects (eg, ELSA-GEN, BiomarCaRE, MedTecHTA, DEXHELPP, EUthyroid, FORECEE, MDS-RIGHT, RECETAS, CORE-MD, EUREGIO-EFH, CIDS, OnCoVID, 4D PICTURE, CATALYSE, EUCAPA, PREMIO COLLAB). He teaches HTA, health economics, modeling, epidemiology, causal inference and target trial emulation, and data and decision science for academia, industry, and health authorities in Europe, North and South America, and Asia. He directs the Continuing Education Program on Health Technology Assessment & Decision Sciences (htads.org).
He has served as member of the ISPOR Directors Board and as president of the Society for Medical Decision Making (SMDM). He is a leadership member of the ISPOR Personalized/Precision Medicine SIG, a member of the Latin America Consortium Advisory Committee of ISPOR, and co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force. He is a member of the Oncology Advisory Council and the National Committee for Cancer Screening of the Austrian Federal Ministry of Health.
He has authored more than 400 publications (>30,000 citations, H index >80), and is editor of the European Journal of Epidemiology. Further information Internet: http://htads.org, umit-tirol.at/dph, hsph.harvard.edu/uwe-siebert, Twitter: @UweSiebert9, LinkedIn: uwe-siebert9.
Case Studies
Oncology Case Studies
These case studies pertain to the evaluation and implementation of health technologies through health technology assessment (HTA) in different healthcare systems. It discusses the factors that influence the recommendation outcomes, efforts to make evaluations more efficient and proportionate, challenges in dealing with confidential information, and the importance of transparency and collaboration in HTA processes.
CS15: Experience of the Implementation of a Payment for Performance Based on a Risk Sharing Agreement Scheme in Argentina: Ribociclib in HR+/HER2- Advanced or Metastatic Breast Cancer Patients
5:30PM - 5:45PM
Cesaroni S1 , Lew D 2 , Manzi M 3 , Fernandez L 3 1 Centro Médico Ceiba, buenos aires, Argentina, 2 Centro Médico Ceiba, Olivos, Argentina, 3 Unión Personal, Buenos Aires, Argentina
Problem Statement: Payment for Performance based on a risk sharing agreements (PBRSAs) are tools that improve access to innovative drugs, especially when there is uncertainty. They link the price of a treatment to the results obtained in clinical practice.
Due to the clinical uncertanty observed with ribociclib in the treatment of patients with HR+ and HER2- advanced or metastatic breast cancer, an PBRSA was implemented.
Description: Based on the published evidence, the eligibility criteria and the thresholds for reimbursement were defined. The End Points used to define reimbursement was Progression Free Survival.
Follow-up meetings with all the parties involved were realized every three months.
Based on the RWD, the Pharmaceutical Company reimbursed the money of the units administered to the patients who progressed, presented toxicity or died before exceeding the defined thresholds.
The 46 patients who received ribociclib in combination with a non steroidal aromatase inhibitor or fulvestrant during the first year of PBRSA implementation were included in the analysis.
At month 12, 6 out of the 46 patients were regarded as treatment failures (3 disease progression, 2 deaths and 1 withdrawals due to toxicity), Direct costs of ribociclib using a traditional payment scheme plus an PBRSA compared to the traditional purchase-only scenario were 7.94% lower.
Lessons Learned: This work demonstrated that it is feasible to implement an PBRSA in Argentina, being essential for its realization a strategic association between the stakeholders involved.
Stakeholder perspective:
CS13: Rocky Road to the Acceptance of Pathological Complete Response (PCR) as Predictor of Improved Disease-Free Survival by HTA Agency in Czech Republic
5:00PM - 5:15PM
Motylova S 1 , Novák J 1 , Dolezel J1 , Pour M2 , Krihova K2 , Skalicky D2 1 ROCHE, s.r.o., Prague, 108, Czech Republic, 2 ROCHE, s.r.o., Prague, Czech Republic
Problem Statement: As early stage intervention with curative intent, the neoadjuvant treatment (NAT) of HER2-positive breast cancer with pertuzumab (P) in combination with trastuzumab (H) lacks mature survival data; hence, it represents excellent example of disconnection between different demands of regulatory and HTA agencies. In line with that, Czech HTA agency (SUKL) initially refused predictive value of pCR for survival and concluded that the evidence provided was insufficient to prove clinical efficacy of P+H combination compared to single-agent therapy in the (neo)adjuvant setting.
Description:
Lessons Learned:
Stakeholder perspective:
CS16: Improving Access to Cancer Therapeutics Through Stakeholders Engagement: A Brazilian Experience
5:45PM - 6:00PM
Nazareth Aguiar P
Problem Statement: Barriers to access to new health technologies in Oncology are not limited to budget issues, a shared decision-making guided by evidence-based medicine plus health economics can improve patients’ access and prevent inequities, such as judicialization.
Description: In line with the 2022-2023 Top 10 HEOR Trends established by ISPOR, Oncoclínicas Group, which is the Brazilian largest private healthcare provider in Oncology, has developed a Medical Access Support Project. In this initiative, a group of specialists receive and analyze all cases with potential barriers to access before sending the claim to the private health insurance. Theses experts review each individual case and raise not only evidence-based medicine data, but also the magnitude of clinical benefit, precision oncology issues, as well as health economics. After this deep analysis, the best individualized management is established for each individual case and, subsequently, there is a roundtable discussion for the shared decision-making between the payer, the health care provider, and also the patient's health care team.
Lessons Learned: The program was established in May 2022. The average time for the first response is 1.72 days and the average time for completing the review process is 2.15 days. The Medical Access Support Project has analyzed 238 cases with regulatory constraints (of which 171 were high-cost regimens) and achieved a rate of treatment authorization of 64% (61% of approval among high-cost regimens). Moreover, considering the 36% of cases in which there was a negative response from the payer, only 6% went to the court. Real-world evidence studies are foreseen in the future of the project.
Stakeholder perspective: Payer, provider, and industry.
CS14: Economic Modelling for Early Decision Making: Opportunity or Further Uncertainty
5:15PM - 5:30PM
Xin Q1 , Chen Y 2 , Pandit U3 , Muszbek N3 , Hawkins N3 , Linsell L2 , Remak E2 , Torlinska B3 1 Visible Analytics, Oxford, UK, 2 Visible Analytics, Oxford, OXF, UK, 3 Visible Analytics, Oxford, Oxfordshire, UK
Problem Statement: Early decision-making is crucial for pharmaceutical/devices companies when developing oncology treatments given the substantial monetary investment required to conduct clinical trials and bring them to market. Understanding the key drivers of future value and data requirements is vital for better decisions.
Description: To facilitate early decision-making, a simplified, flexible, user friendly early economic model and pricing tool in R and rShiny was developed and tested in three case studies (optimistic, pessimistic, neutral). This tool was designed to analyse early phase clinical data from patients with advanced oncology diseases with the aim of predicting economically justifiable drug prices (EJPs). It used a lifetime partitioned survival analysis approach based on overall survival and progression-free survival. Given the limited survival data at early phases, the tool accommodates either Kaplan-Meier curves or one to two survival data points. The ‘flexsurv’ packages from R 4.3.0 were used for the survival analyses. The case studies were selected from recent advanced oncology technology assessments by the National Institute for Health and Care Excellence. EJPs estimated based on Phase I/II trial data were compared with predictions using corresponding Phase III data under different survival curve assumptions. Results showed that the incorporation of external data on comparators, prognostic factors, and long-term survival assumptions not only enhanced the prediction of EJPs but also resulted in improved decision-making and a better understanding of the implications associated with clinical trial designs, including disease severity, treatment lines, combination treatments, and patient demographics.
Lessons Learned: There are significant uncertainties around using early clinical trial data in predicting future value in advanced oncology. Early economic modelling of EJP could reduce uncertainties, provide companies with valuable insights for the evidence generation plans, help with go/no go decisions from a cost-effectiveness perspective.
Stakeholder perspective: The case study was carried out from an industry perspective.
Podium Sessions
Innovations in Modeling for Health Technology Assessment
Digital Conference Pass
This podium session will feature cutting-edge health tech assessment, AI economic modeling, Simpy's patient simulations, and open-source Alzheimer's evaluation framework.
Moderator
Thor-Henrik Brodtkorb, PhD
RTI Health Solutions, Ljungskile, O, Sweden
Thor-Henrik Brodtkorb, PhD, is executive director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 12 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision-analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and ISPOR.
At RTI-HS, Dr. Brodtkorb leads the development of cost-effectiveness, cost-utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
P4: Testing SimPy, a Library for Patient-Level Simulations in Python: An Application to Resource Management in Healthcare Systems
5:45PM - 6:00PM
Felizzi F
OBJECTIVES:
The NICE Decision Support Unit presented a Technical Support Document report to carry out patient-level simulations for cost-effectiveness modeling in R, VBA and Simul8. We tested the SimPy Python library and discuss its use. In the light of the bottlenecks which emerged after the COVID-19 pandemic, we applied SimPy to a real-world scenario of an ophthalmic clinic. METHODS:
The SimPy library enabled the creation of processes defined by Python generator functions. In SimPy one can DEFINE resources, such as specialists, available slots for visits, and create instances of these resources with their capacity. One can REQUEST or RELEASE resources, such as the arrival and dismissal of patients, PRIORITIZE resources, CAPACITY management, i.e. the dynamic evolution of additional resources being provided on a bespoke manner and SYNCHRONIZATION between resources. It provides tools for collecting and analyzing STATISTICS during the simulation, such as data on resource utilization, waiting times, queue lengths, or any other relevant metrics to evaluate and optimize healthcare processes. RESULTS:
Unlike R, Python is an object-oriented programming (OOP) language. The key feature of encapsulation helps define methods in resource classes to handle interactions such as a patient being assigned to a bed. These interactions can be encapsulated within the objects, making the simulation logic more intuitive and maintainable. Inheritance and polymorphism enable capturing common characteristics, while allowing for specialization and the uniform treatment of objects of different resources with similar behavior. OOP promotes modularity, reusable classes representing common healthcare resource patterns can be combined to build more complex simulations. CONCLUSIONS:
The use of SimPy provided to be a very handy and easy to learn Python library when performing a patient-level simulation. In the light of the popularity of Python, the health economics modeling community should explore the advantages of embracing its use.
P3: Open-Source Model Framework for Health-Economic Evaluation of Early Treatment in Alzheimer's Disease
5:30PM - 5:45PM
Handels R 1 , Wimo A2 , Skoldunger A2 , Tate A3 , Winblad B2 , Silvertand D1 , Nguyen L1 , Jönsson L2 , Grimm S4 , Aye S5 , Herring W6 1 Maastricht University, maastricht, LI, Netherlands, 2 Karolinska Institutet, Stockholm, Sweden, 3 Karolinska Insitutet, Solna, Sweden, 4 Maastricht University Medical Center, Maastricht, Netherlands, 5 K, Stockholm, Sweden, 6 RTI Health Solutions, Research Triangle Park, NC, USA
OBJECTIVES: Recent drug developments in Alzheimer's disease (AD) have increased attention for decision-analytic models to assess their value. Transparency and credibility of decision-models are key for their results to be used by decision-makers. Relatively short-term trial evidence requires assumptions on long-term treatment effect, which drives uncertainty.
We aim to develop an open-source AD decision-model framework for the health-economic evaluation of emerging AD treatments.
METHODS: A previously developed Markov model (MMSE-based states: mild cognitive impairment (MCI), mild, moderate, severe dementia and death) was rebuilt in R and internally validated by replicating its published outcomes (maximum relative difference<0.6%). The model was updated with new features including treatment discontinuation, European country/region-specific mortality, costs and utilities, and probabilistic analyses on natural disease history parameters.
A hypothetical treatment with 25% reduction in 70-year old early AD patients (progression rate from MCI to mild dementia and mild to moderate dementia) with 10% treatment discontinuation and 5% waning per year, maximally treated for 7 years was compared to standard care. We calculated the maximum price at which treatment would be cost-effective and performed sensitivity analyses on net health benefits. The model is freely available via IPECAD (www.ipecad.org/open-source-model), including coding comments and a deterministic spreadsheet.
RESULTS: Early AD treatment was cost-effective at €4444, €3170, €4692, €5385, €3943 price per year per person in British Isles and in East, North, South and West Europe respectively (WTP=€40,000/QALY).
Net health benefit was mainly sensitive to treatment costs, treatment duration, treatment effect on dementia onset and starting age.
CONCLUSIONS: We developed an accessible open-source model framework for health-economic evaluation of early treatment in AD. The sensitivity to natural progression and age reflects the importance of predicting treatment response for new treatments. Planned features include budget impact module, online interface and individualized progression to allow evaluation of individualized treatment per country.
P2: NICE's Pathways Pilot: Incorporating Disease-Specific Reference Models Into Health Technology Assessment in England
5:15PM - 5:30PM
Brooke A 1 , Dunning L2 , Bell E1 , Watson I3 , Dawoud D4 , Bouvy J5 1 NICE, Manchester, UK, 2 NICE, London, LON, UK, 3 National Institute for Health and Care Excellence (NICE), London, UK, 4 National Institute for Health and Care Excelllence, London, LON, UK, 5 National Institute for Health and Care Excellence, London, LON, UK
OBJECTIVES: Like other health technology assessment (HTA) bodies, NICE is seeing an increasing number of technologies being appraised in the same disease area, creating complex pathways with multiple sequential decisions and multi-comparator decision spaces. HTA bodies need to adapt their methods to meet the challenges of providing robust, useful, and timely guidance with ever increasing demand. NICE is developing refined appraisal processes that are proportionate to different needs. Pathways is one approach, built around a core model that spans a disease pathway and multiple technologies.
METHODS: NICE’s pathways pilot explores the development of a core model to appraise treatments in the systemic treatment pathway for renal cell carcinoma. The pilot was informed by a targeted review of the literature, a focus group, and engagement sessions with key stakeholders. The pilot process is being monitored and refined.
RESULTS: The review identified that 43 percent of HTA guidance produced by NICE covers only ten disease areas. Engagement sessions established a range of procedural, methodological, and operational challenges for disease-specific reference models, including the complexity of analysis, the reliance on partition survival modeling in cancer, and the availability of evidence. However, it was clear that this is an evolving area of interest for HTA bodies. The use of a single model promises to increase transparency and reduce structural uncertainties, but concerns exist that these models may accentuate challenges common to all economic evaluations. The pathways pilot explores whether technological developments and real-world evidence can address these challenges whilst producing robust guidance.
CONCLUSIONS: The pathways pilot can pave the way for dynamic changes in HTA. It can create efficiencies by incorporating novel technologies into disease-specific models, with economic and clinical data updated as required. Using a pilot to address the challenges allows solutions to be found, and future directions to be considered collaboratively.
P1: Automating Economic Modelling: A Case Study of AI's Potential With Large Language Models
5:00PM - 5:15PM
Reason T1 , Rawlinson W 2 , Malcolm B3 , Klijn S4 , Langham J5 , Gimblett A2 1 Estima Scientific Ltd, South Ruislip, LON, UK, 2 Estima Scientific Ltd, London, UK, 3 Bristol Myers Squibb, Middlesex, LON, UK, 4 Bristol Myers Squibb, Lawrence Township, NJ, USA, 5 Estima Scientific Ltd, London, LON, UK
OBJECTIVES: The potential to integrate large language models (LLMs) such as GPT-4 in script extraction and generation could revolutionise the creation and deployment of economic models. The aim of this study was to assess the validity of a partitioned survival model produced by GPT-4 against a published model, comparing treatments for advanced or metastatic non-small cell lung cancer (NSCLC) for patients who had disease progression after platinum-based treatment and had not received prior immunotherapy (CTLA4 or PD-[L]1 inhibitors).
METHODS: GPT-4 was instructed by providing prompts to replicate an existing 3-state partitioned survival model for NSCLC by modifying a generic shell R script. Data for the model, assumptions, and choice of parametric models to fit OS and PFS were provided to GPT-4, extracted from the publication. The output of the AI generated model was compared with the original published model to evaluate the validity and accuracy.
RESULTS: GPT-4 successfully adapted a shell script, extracting data from pre-generated tables, to produce a 3-state partitioned survival cost-effectiveness model script for NSCLC. The resultant script compiled with no edits by a human modeller. Total costs and total QALYs for the AI-generated model were within 1-10% of the outcomes in the publication and observed differences were primarily explained by model inputs such as the data quality and availability (digitised survival curves vs individual patient data, and an unpublished input value) rather than inaccuracies in the AI-generated model.
CONCLUSIONS: The final model closely resembled the published version and yielded similar outputs, substantiating the reliability of the AI-generated model. This study offers evidence for the incorporation of LLMs, such as GPT-4, in the automation of economic modelling, specifically in script generation. Future research is necessary to corroborate results and further explore the potential of AI to streamline and enhance the quality of economic modelling.
Breakouts: IP, WS, & OBS
Charting a Greener Future: Should Environmental Impact Factor Into HTA Evaluations?
Digital Conference Pass
Level: Introductory
ISSUE: Tackling the climate crisis is an international priority. Health care systems are major emitters, and whilst many national and international targets are in place, health technology assessment (HTA) has not yet been fully explored as a vehicle for change.
HTA agencies have expressed interest in capturing environmental effects, and stakeholders believe HTA actors can enable change. Various case studies and commentaries exist which propose tentative methods for including effects on the environment into HTA, but many of these highlight evidential and methodological roadblocks. Several questions must be explored: Is it appropriate to capture environmental effects in HTA? If so, how can this be operationalised? Where does the balance of responsibility between the manufacturer and the health care system lie? What are the intended and unintended consequences of including environmental effects in HTA? And crucially, how much health gain are we willing to trade for a reduction in environmental effects? This panel will debate the above questions, drawing on preliminary research and the panel’s experience of the methodological, operational and ethical complexities of changing the shape of traditional HTA in this way. The panel will critically appraise the likely consequences, and tackle the key issue of who really should pay for ‘green’ pharmaceuticals – health systems (and ultimately patients via reduced health gain), manufacturers, or wider stakeholders.
OVERVIEW: Following a brief introduction to the issues by the moderator (4 mins), the panel will debate the future of sustainability within value assessment, including scope, methods and evidence. Panellists will each speak for 12 minutes, providing their perspectives on the future of environmental impacts and HTA, including appropriateness, methods, and how to overcome the challenges. 20 minutes will be reserved for audience discussion.
Moderators
Grace Hampson, MSc
Office of Health Economics, London, United Kingdom
Grace is an economist with expertise in the methods and processes of value assessment, reimbursement and healthcare decision making. Her current research interests include interactions between health technology appraisal and novel payment mechanisms, particularly in relation to innovative technologies such as cell and gene therapies and new drugs to tackle Antimicrobial Resistance. Grace leads OHE’s Policy Organisation and Incentives in Health Systems research team.
Panelists
Isabell Carmela Crasto De Stefano, MA
CHIESI FARMACEUTICI, Parma, Italy
Advocating for Respiratory Health & Societal Progress
Passionate about driving positive change, I strongly focus on advancing political and social awareness in the realm of respiratory diseases.
My mission centers around the recognition that well-crafted international and national respiratory policies can be instrumental in saving lives, reducing prevalence, and elevating the overall health and quality of life for those impacted by respiratory conditions.
At the heart of my approach is a commitment to patients' wellbeing. By championing informed and evidence-based advocacy, I aim to inform critical decision-making processes and advise policies that drive positive societal outcomes.
Operating with a deep-rooted conviction in the principle of "shared value," I seek to foster constructive partnerships and collaborative efforts within multi-stakeholder networks. Embracing co-creation and synergies, I work tirelessly to maximize the societal impact of our initiatives and ensure a holistic approach to tackling respiratory challenges.
With a track record of successful engagements on both national and international levels, I am driven to make a lasting impact and contribute to the advancement of respiratory health worldwide. Join me in this journey as we create a healthier, more resilient future for all.
Nadine Henderson, MSc
Office of Health Economics, London, LON, United Kingdom
Nadine Henderson is a Senior Economist at the Office of Health Economics, London, whose research interests centre on issues around the methodology and process of Health Technology Assessment. She has evaluated the appropriateness of HTA methods to assess orphan and ultra-orphan medicines, gene therapies, and tumour-agnostic therapies. She has previously published on the topic of incorporating severity in HTA and is currently investigating how the pharmaceutical industry can become more sustainable and the scope for incorporating environmental impact in HTA. Her other research areas include measuring and valuing health-related quality of life using qualitative methods.
Saskia Knies, PhD
Zorginstituut Nederland, Diemen, NH, Netherlands
Saskia Knies, PhD is senior advisor health economics and personalised medicine at the National Health Care Institute in het Netherlands (Zorginstituut Nederland; ZIN). In addition, till recently she was the coordinator of the research network HTA, which is a collaboration between ZIN and Erasmus University Rotterdam and Utrecht University. She holds a MSc in Health Science Research – specialisation HTA from Maastricht University and a PhD in health sciences also from Maastricht University. Due to her experience as pharmacoeconomic assessor she has extensive experience in using economic evaluations for reimbursement decision making. Saskia’s research interest are focused on HTA methods development to support healthcare decision making especially concerning economic evaluations, transferability of (health economic) evidence and on international comparisons. Saskia is also involved in the H2020 funded research project HTx. Her main task in all her work is building bridges between research (especially methods development or policy research) and the use of these methods in supporting decision making. Saskia has multiple publications in the field of HTA and pharmacoeconomics. She has been actively involved in different ISPOR activities, among others faculty member of a short course on transferability, member of ISPOR Taskforce on Value of Information, member of the HTA Roundtable Europe, reviewer for Value in Health and presenter in several panels and workshops. Saskia is also a guest senior researcher at the Erasmus School of Health Policy & Management (ESHPM) at Erasmus University Rotterdam.
Challenges and Opportunities in Adopting a Societal Perspective in Health Technology Assessment
Digital Conference Pass
Level: Intermediate
ISSUE: The perspective of an economic evaluation determines which types of outcomes are counted in cost-benefit calculations. Health technology assessment (HTA) agencies typically define a reference case perspective that is applied across assessments to ensure consistency and fairness. Whilst some agencies specify a health sector perspective for their health economic analyses, which counts health outcomes and healthcare costs, others have opted for a societal perspective that includes additional effects, the most common being the impacts on economic productivity.
Proponents of a societal perspective argue that a health sector perspective misses important relevant effects and can lead to HTA agencies not recommending treatments that have positive net social effects. Conversely, a broader perspective raises a different set of practical, methodological and ethical issues relating to the consistency, transparency and robustness of economic assessments. These include determining which aspects of benefit and cost should be included, how they should be valued and concerns about the quality of supporting evidence. In recent years, several HTA agencies have reviewed, and in some cases changed, their approach to perspective in light of these challenges.
OVERVIEW: The moderator will begin the session with a brief introduction and update on NICE’s recent review of its approach to perspective (5 mins). The panel will then explore the key issues around the choice of perspective and provide insights on whether and how a societal perspective should be implemented within HTA (10 mins per panellist). The session will conclude with an open discussion with the audience (20 mins).
Moderators
James Koh, PhD
National Institute for Health and Care Excellence (NICE), Manchester, LAN, United Kingdom
James Koh is a Scientific Adviser at the National Institute for Health and Care Excellence (NICE) and an Honorary Research Fellow at the Centre for Health Economics. His work covers novel science policy and research topics at NICE, including the evaluation of antimicrobials and complex diagnostics and its approach to economic perspective and inequalities in assessments.
James has previously worked in both consulting and academic roles, including as a Research Fellow at CHE between 2017 and 2021, specialising in the development of distributional cost-effectiveness analysis methods to incorporate inequality concerns into health economic analysis. He holds an MSc in Health Economics and PhD in Economics, both from the University of York.
Panelists
G. Ardine de Wit, PhD
National Institute for Public Health and the Environment, Bilthoven, UT, Netherlands
Ardine de Wit is a specialist in the field of Health Technology Assessment, working as senior researcher at the National Institute of Public Health and the Environment and as Professor (endowed) at the Department of Health Sciences of Vrije Universiteit Amsterdam. She has been involved in a great number of economic evaluations, including quality of life assessment, of health care interventions, both in the field of public health and medical technology. She (co-)authored 200 international publications and 60 research reports in these fields. She currently chairs an expert committee that reviews the Guidelines for economic evaluation issued by the National Health Care Institute.
Durgesh Kahol, MSc, MBA, BPharmacy
GlaxSmithKline, Ickenham, United Kingdom
Durgesh has 13 years of experience in the life sciences industry, specialising in commercial strategy and operations of Health Economics, Real World Evidence generation and Market Access. She is currently a Director in Health Outcomes and Access for Oncology at GSK, and has over 10 years experience working in market access and health economics roles across industry and consultancy. Durgesh has extensive experience developing HTA submissions in the UK and has contributed to a range of projects with a global focus.
Douglas Lundin, PhD
Tandvårds- och läkemedelsförmånsverket (TLV), SKÖNDAL, Sweden
Douglas Lundin is chief economist at TLV, the Swedish HTA and reimbursement agency. He has had that position since 2011. He has also worked at the Swedish Competition Authority. He holds a PhD in economics from Uppsala university and has also been a researcher at Center for health economics, Stockholm school of economics.
How to Assess Patient Preferences for Use in Decision-Making Along the Medical Product Life Cycle? Learnings From Patient Preference Studies Across Diverse Disease Domains
Digital Conference Pass
Level: Intermediate
ISSUE: Patient preference studies (PPS) yield evidence about patients' desirability or acceptability of a particular alternative or choice among all outcomes of a particular drug. Such evidence is considered highly valuable to inform decisions along the medical product life cycle such as in unmet needs identification, clinical trial endpoint selection, regulatory benefit-risk assessment, Health Technology Assessment and reimbursement, and clinical decision-making. While the added value is clear, there is lack of case examples and understanding on how the assessment of PPS should occur to robustly inform decision-making. Methodological questions exist regarding appropriate PPS design and conduct, including regarding the development of the research question (product agnostic or not), method selection, patient recruitment, sample size, attribute and level development and qualitative research in this effort, PPS analysis, availability and transparency of PPS data and methods, and stakeholder engagement (incl. patient, clinician, regulatory and HTA bodies).
OVERVIEW: Drawing upon experiences from PPS’ in diverse disease domains, speakers from different stakeholder organisations (University Medical Center Groningen, European Medicines Agency, KU Leuven, EFCCA) will share their views (regulatory, methodological, patient) on how PPS can be robustly designed to provide evidence-based information considered useful by decision-makers. The session will cover both qualitative (interviews, focus group discussions) and quantitative methods (Discrete Choice Experiment, Swing Weighting, threshold technique). Presenters will illustrate how they approached their PPS design such as the development of attributes and levels (such as life expectancy and quality of life-related attributes), the choice of methods and selection of patients and sample size. The session will discuss how patients and patient organizations should be engaged as active research partners in informing key decisions throughout the PPS development and dissemination. This session is intended to augment best-practice insights and potential improvements regarding the design and conduct of PPS aiming to inform clinical trial design, regulatory and HTA bodies.
Moderators
Isabelle Huys, PharmD, PhD
Department of Clinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, VBR, Belgium
Prof. dr. Isabelle Huys is full professor at the University of Leuven (Belgium). She leads the research group on Regulatory Sciences at the Faculty of Pharmaceutical Sciences, embedding the Patient Evidence and Preference Center (PPRC). Her research focusses on market access of medicines and therapies, with particular attention to the inclusion of the voice of the patient in drug development, evaluation, market access and usage. In addition, ethical, legal and social issues (ELSI) are studied in an interdisciplinary context.
Panelists
Maria Stella De Rocchis, MSc
European Federation of Crohn's & Ulcerative Colitis Associations (EFCCA), Brussels, WBR, Belgium
Maria Stella De Rocchis is Project Manager at European Federation of Crohn's and Ulcerative Colitis Associations, representing 46 national Crohn's and Ulcerative Colitis patient organisations. She was an active research partner in a qualitative and quantitative preference research among patients with Inflammatory Bowel Diseases, where she contributed to the development of the research questions, the selection and description of the attributes and levels, piloting, patient recruitment, and survey dissemination. Maria Stella has a Master's degree with Honours, Psychology of Communication and Marketing.
Douwe Postmus, PhD
University of Groningen, University Medical Center Groningen, Groningen, GR, Netherlands
Dr. Douwe Postmus is working as an assistant professor at the Department of Epidemiology of the University Medical Center Groningen, The Netherlands. His research focuses on applying statistical and decision analysis techniques to decision making problems in health care, including regulatory, reimbursement, and shared decision making problems. He has an h-index of 26, co-authored 78 publication(s) receiving 1703 citation(s).
Elise Schoefs, MSc
Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium
18:00 - 19:00
Women in HEOR: Networking and Meet the Speakers Event
Meet the Women in HEOR Session speakers and mingle with other women in HEOR and "cool guy allies" at the ISPOR Booth!
Networking Reception
Taking place in the Exhibit Hall
Wed 15 Nov
7:00 - 8:00
Morning Coffee Service
7:00 - 13:00
Registration Hours
8:00 - 9:00
Case Studies
HTA Case Studies II
These case studies involve the evaluation and implementation of health technologies through health technology assessment (HTA) in different healthcare systems. They discuss the factors that influence the recommendation outcomes, efforts to make evaluations more efficient and proportionate, challenges in dealing with confidential information, and the importance of transparency and collaboration in HTA processes.
Moderator
Sorrel Wolowacz, PhD
RTI Health Solutions, Manchester, United Kingdom
Sorrel Wolowacz, PhD, is Head of European Health Economics at RTI-HS, with 22 years of experience in health economics research and consulting. Her research focuses primarily on economic modelling, health utility estimation, observational studies, and health technology appraisal submissions. Dr. Wolowacz is a member of the editorial board for the Journal of Comparative Effectiveness Research and was co-chair of the ISPOR Good Research Practices Task Force addressing Measurement of Health State Utility Values for Economic Models in Clinical Studies and is a member of the ISPOR Oncology Special Interest Group.
CS18: Streamlining Health Technology Assessment: Fast-Tracking NICE’s Technology Appraisal Processes
8:15AM - 8:30AM
Mayers C 1 , Kenny G2 1 National Institute for Health and Care Excellence (NICE), Ashton-under-Lyne, UK, 2 National Institute for Health and Care Excellence (NICE), Manchester, Greater Manchester, UK
Problem Statement: Ahead of the 2022/23 business year, projections for 2023/24 indicated a 20% increase in evaluations coming through the NICE technology appraisal programme. Responding to the predicted pipeline, NICE explored ways to make its evaluations more efficient and proportionate. One aspect of this was reconfiguring NICE’s approach to cost comparison appraisals. The cost comparison process, developed in 2017, was designed for the most cost-effective new treatments, aiming to provide quicker access for patients through faster implementation. However, it did not meet its full potential in terms of speed and efficiency. In 2022/23, the process was redesigned to make it faster and more proportionate. This work was part of NICE’s Proportionate Approach to Technology Appraisals (PATT).
Description: NICE’s cost comparison process was introduced for technologies likely to provide similar or greater health benefits at similar or lower cost than technologies already recommended in technology appraisal guidance for the same indication. This process was the same length as the standard process and required a similar amount of internal and external resource. The key routing decision was set half-way through the process. In 2022/23, the process was redesigned. By seeking early clinical input and making the routing decision before an evaluation starts, the key stages could be shortened. Adopting a test-and-learn approach, the process was trialled on live evaluations as NICE continually engaged with stakeholders to refine the process.
Lessons Learned: The new process for cost comparison appraisals is 30% faster than the 2017 process. Implementing faster, light-touch processes for simple evaluations demonstrates NICE’s commitment to producing useful, useable, and timely guidance for patients and healthcare providers. Demonstrating the effectiveness of this streamlined approach for cost comparisons allowed further streamlining of the standard process for a wider range of medicines. Data collected on resource savings allows for further refinement of the process.
Stakeholder perspective: Government-affiliated.
CS19: What Lessons Can Pharmaceutical Companies Learn From the First Published Joint Clinical Assessment (JCA) Report?
8:30AM - 8:45AM
Robert A 1 , Pardalidis V2 1 PRMA Consulting Ltd., Fleet, Hampshire, UK, 2 PRMA Consulting Ltd., Athens, Athens, Greece
Problem Statement: EUnetHTA joint clinical assessments (JA) are jointly produced by EUnetHTA partners in different European countries with the aim of substituting parallel evaluations of clinical data with a single harmonized process. Analysis of the first published report provides an opportunity for drug manufacturers to understand what additional factors should be considered with reference to JCA evidence requirements.
Description: The first published JCA report assessed the relative clinical effectiveness and safety of Optilume, a paclitaxel-coated urethral balloon for the treatment of urethral strictures in adult males. The project was managed by Zorginstituut Nederland (ZIN; The Netherlands) and the assessment team consisted of members of Haute Autorité de Santé (HAS; France), and the Austrian Institute for Health Technology Assessment (AIHTA; Austria). The process began on 4th October 2022 with a scoping phase, followed by an assessment phase beginning on 30th January 2023, and ending with publication of the final report on 6th June 2023.
Lessons Learned: The PICO survey resulted in 3 consolidated PICOs that only differed from each other in terms of comparator. The assessment team found that of the three pivotal studies submitted by the manufacturer, no study individually addressed the PICO 1, 2 or 3 question, but one sufficiently addressed elements of PICOs 1 and 2 collectively. Uncertainties raised by the assessment team included the risk of bias which was considered high for all outcomes due to single blinding, the fact that the trial wasn’t conducted in Europe, the lack of results for drug-related adverse events, and a lack of clear explanation for handling of missing data.
Stakeholder perspective: Outside of methodological or statistical concerns often voiced at national HTA level, drug manufacturers should also be mindful of aligning trial design parameters fully with the expected PICO framework, and ensuring sufficient European-level recruitment in the pivotal trial.
CS20: Updating NICE’s Methods on Confidentiality Handling
8:45AM - 9:00AM
Wilkes S 1 , Hughes M1 , Landells LJ1 , Spanswick C1 , Prescott C2 1 National Institute for Health and Care Excellence, Manchester, Greater Manchester, UK, 2 National Institute for Health and Care Excellence, Manchester , LAN, UK
Problem Statement: In 2022, the International Committee of Medical Journal Editors (ICMJE) announced it no longer considers data published by HTA agencies to be duplicate publication. This prompted the National Institute for Health and Care Excellence ( NICE ) to reconsider its policies on confidential information in their evaluations, particularly “academic-in-confidence ”. This work formed part of NICE’s Proportionate Approach to Technology Appraisals (PATT).
Description: NICE critically looked at the amount and types of confidential information it receives in its submissions as well as the administrative burden associated with confidentiality marking . Through this, possible barriers to removing “academic-in-confidence” data were identified and considered. W e engaged with pharmace utical industry representatives and other NICE stakeholders to gather insight into potential barriers and possible solutions . NICE also collaborated internationally with other Health Technology Assessment (HTA) bodies to create an aligned approach to confidentiality. In April 2023, NICE released a joint position statement with the Canadian Agency for Drugs and Technologies in Health ( CADTH ) and the Institute for Clinical and Economic Review ( ICER ) outlining their joint approach to confidential information including information pending publication. Alongside this, NICE released interim methods as part of a proportionate approach to technology appraisals replacing the “academic-in-confidence” and “commercial-in-confidence" confidentiality categor ies with a single confidential category and outlining the types of data that could and could not be marked confidential. NICE is currently assessing the impact of the position statement and interim methods on the redaction needs of stakeholders , transparency of HTA and administrative burden of confidentiality.
Lessons Learned: There is a balance between providing patients, clinicians and the public with timely , useful, usable guidance while protecting industry’s interests. The ICMJE statement, international HTA collaboration and NICE’s interim methods demonstrate progress towards pragmatic a l l y increasing transparency in HTA. Further lessons learnt and barriers encountered will be presented in the final poster .
Stakeholder perspective: Government-affiliated
CS17: A Yes or a No? Decicive Factors Behind Recommendations Made by the Danish Health Technology Council
8:00AM - 8:15AM
Liljenberg D
Problem Statement: In 2023 the Danish Health Technology Council (DHTC) published health technology assessments (HTA) on, among others, sensor-based glucose monitors for adults with type 1 diabetes and the use of artificial intelligence (AI) in colonoscopy. The HTAs led to a recommendation for and recommendation against a national implementation of the technologies, respectively. This abstract summarizes factors that were decisive for the outcomes of the recommendations.
Description: DHTC methods dictate that four perspectives are to be examined in HTAs of health technologies: clinical effectiveness and safety, the patients’ perspective, organizational implications, and health economics. Covering these four perspectives, the Council is to decide if a health technology should be recommended for national implementation. By comparing two HTAs, with a recommendation for and against national implementation, respectively, decisive factors for the recommendation outcomes are identified.
Lessons Learned: DHTC emphasized the following elements as decisive for their recommendations:
The use of glucose monitors:
Leads to pronounced positive effects on several clinical outcomes
Has compelling, positive consequences for patients, with potential to improve patients’ treatment satisfaction, quality of life, and insight into treatment and disease
Is supported by a solid evidence base
Is appraised by clinicians as a valuable tool for improving quality of treatment and communication with patients
In contrast, the use of AI:
Might constitute a risk to patient safety, if leading to the collection of unnecessary tissue samples with the risks this entails
Has not been evaluated with respect to patient-relevant outcomes
Is supported by an inadequate evidence base
Does not usefully support clinicians’ decision-making, who often opt out of using the AI-function
Stakeholder perspective: DHTC bases its recommendation on a consideration of multiple factors, where patient-centeredness, perceived value amongst clinicians, and the solidity of the evidence base supporting the use of the technology were emphasized as pivotal for the outcome of the national recommendations.
Breakouts: IP, WS, & OBS
Long-Term Tracking of Clinical Effectiveness, Safety, and Value of Gene Therapies and Implications for EU Joint Clinical Assessment: Multistakeholder Perspectives From Patients, Manufacturers, and Payers
Digital Conference Pass
Level: Introductory
PURPOSE: Demonstrating long-term (LT) effectiveness, safety, and value of gene therapies necessitates linking data from diverse data sources, safeguarding data quality, privacy, and security, and minimizing burden to patients, providers, and manufacturers. When the LT value of gene therapies is demonstrated, it also creates a need to revisit past reimbursement decisions and adjust them into the future. From a multi-stakeholder’s perspective, this workshop will use real-time polling with an audience discussion about the opportunities, challenges, and policy implications, especially for the EU Joint Clinical Assessment (JCA).
DESCRIPTION: Gene therapies disrupt traditional treatment paradigms. The transformative nature of a potentially one-time treatment as the potential “next-gen therapy” creates the need to revisit how LT clinical effectiveness, safety, and value are demonstrated.
From a patient advocacy perspective, a use case by the World Federation of Hemophilia Gene Therapy Registry will demonstrate the value of incorporating technologies to maximize patient engagement in reporting LT data while minimizing the burden on patients. This creates the need to revisit the reliability and validity of current outcomes from a patient perspective and may re-shape regulatory and health technology assessment (HTAs) policies. From a manufacturer’s perspective, challenges of collecting consistent LT real-world data (RWD) to support HTA reimbursement decisions for gene therapies will be discussed. Use cases of unique partnerships will illustrate approaches to improve real-world evidence generation using existing data infrastructures. From a life-science service organization’s perspective, leveraging technologies to integrate RWD from active registries, patient-reported outcomes, electronic medical records, and claims data to support regulatory-grade RWE generation will be presented. From a payer/HTA’s perspective, emerging LT RWD from gene therapies also creates the need to revisit past reimbursement/financing decisions and adjust these decisions and other relevant policies for the future. New methodologies will be needed and adapted to the unfolding EU JCA.
Discussion Leaders
Martina Garau, MSc
Office of Health Economics, London, United Kingdom
Discussants
Ruth Kim, PharmD, MPH
Pfizer Inc, New York, NY, USA
Ruth Kim is an intrapreneur in developing unique launch plans for pharmaceutical companies. During her tenure in industry, she successfully led a team launching multiple immuno-oncology assets, with extensive publications in oncology. Ruth established companywide real-world evidence and epidemiology functions at Medivation and Bristol Myers Squibb. Now at Pfizer, she is the Health Economics and Outcomes Research (HEOR) Therapeutic Area Leader for Rare Disease and Internal Medicine.
Ruth is a pharmacist by training with a master’s in public health. She completed her post-doctoral fellowship in HEOR and Epidemiology focusing on Haemophilia and Hepatocellular Carcinoma. Prior to joining industry, she was an adjunct professor in physical chemistry, pharmacoeconomics and pharmacy technics.
Naja Skouw-Rasmussen, MScGeo
European Haemophilia Consortium, Brussels, Belgium
Naja Skouw-Rasmussen has a long-standing involvement in the bleeding disorder community with ten years of experience on the board of directors at the Danish Haemophilia Society (DHS). She served as member of the European Haemophilia Consortium (EHC) Steering Committee from 2016-2021 and was founding member and Chair of the EHC Women and Bleeding Disorder Committee from 2017-2021. She has played an active role in various projects like the Danish project on telemedicine, a youth project in India and, more recently, in developing the European Principles of Care for Girls and Women with a Bleeding Disorder.
Ms. Skouw-Rasmussen holds a Master of Science in Geography from the University of Copenhagen and a Master of Science in Development Studies from SOAS in London. She has worked with the civil society in the Danish National Council of Volunteering and managed professional networks for corporate leaders and managers.
Ms. Skouw-Rasmussen works as a consultant for the EHC driving the EHC Think Tank forward currently in the thematic areas of health data registries, patient agency, access equity and future care pathways. As a volunteer she is devoted to the women and bleeding disorder agenda and serves as patient representative at the EAHAD women and bleeding disorder working group, and work with the Danish Haemophilia Society.
Ying Tabak, PhD
UBC, Blue Bell, PA, USA
Dr. Tabak leads development and execution of RWE/HEOR research strategies at UBC. She has extensive leadership and research experience across RWE, HEOR, epidemiology, biostatistics, and data science. She has numerous podium presentations across a range of research conferences. She served as lead or senior author for the majority of her publications with >6,000 citations. She also served as a reviewer for NEJM, JAMA, and other prominent journals. She will be a panelist for this session.
Longitudinal Qualitative Research: Methodological Approaches and Potential Applications to Patient Centered Outcomes Research
Digital Conference Pass
Level: Intermediate
PURPOSE:
Longitudinal qualitative research (LQR)—where the same participants are interviewed multiple times and data analyzed longitudinally to characterize changes over time—can provide unique insights in clinical trial and real world settings, such as characterizing meaningful treatment benefit for the population under study and understanding disease trajectory to describe the course or progression of disease. However, LQR is underutilized and/or poorly reported in many domains of patient outcome research. Understanding the current state of LQR methods and potential applications can help guide the expansion of this methodological approach in the health outcomes research domain. This workshop will provide insights on benefits and potential challenges of LQR and provide opportunities to learn about methodological approaches for collecting and analyzing longitudinal qualitative data in clinical trials and real-world studies. DESCRIPTION:
After an introduction of LQR purpose and current methodological approaches, the speakers will present potential applications and examples of LQR in clinical trial and real-world settings and address resulting contributions to patient centered outcomes research in each case study. Introduction to LQR, potential applications and value to stakeholders (10 min) Designing LQR studies and practical challenges (10 min) Potential real-world LQR opportunities using ovarian and breast cancer cases studies as examples (10 min) Case study on the use of longitudinal mixed methods approach to assess treatment benefit in Migraine in the context of a clinical trial (10 min) The audience will be invited to further the knowledge gained by developing a list of potential applications for LQR to patient outcome research, with stated objective(s) and possible challenges (20 min). At the end of the workshop, participants will: Understand the benefits of LQR data and their value in drug development and real-world settings Understand methodological approaches to implement LQR and potential challenges Discuss potential applications and outcomes of LQR in patient outcome research
Discussion Leaders
Carla Dias Barbosa, MSc
Evidera, London, United Kingdom
I’m a Senior Research leader on the Patient-Centered Research team at Evidera, a PPD business, responsible for developing exit and embedded clinical trial interview study business. I have more than 20 years’ experience in conducting health outcomes research studies, including instrument development and psychometric evaluation, design, and implementation of clinical outcomes assessment (COA) endpoint strategies for clinical programs, quantitative and qualitative COA studies, and literature reviews. I also have extensive experience designing, implementing, and analyzing exit and embedded clinical trial interview studies using novel approaches such as mixed-methods research (MMR) and longitudinal qualitative research (LQR) approaches. This work has been performed in a wide variety of disease areas, including skin diseases, pain, endocrinology, musculoskeletal disorders, respiratory diseases, oncology, rare diseases, and pediatric diseases. I chaired the International Society for Quality-of-Life Research (ISOQOL) Mixed Method Research Special Interest Group from 2018 to 2020, and currently lead an ISOQOL program that aims to promote the use of longitudinal qualitative research in health outcomes research and defining best practice methodologies.
Discussants
Kimmie McLaurin, MS
AstraZeneca, Adamstown, MD, USA
Kimmie McLaurin is the Oncology Outcomes Research lead for GU and GYN tumors at AstraZeneca. She leads the global team responsible for generating real-world evidence that support decision making to improve patient care. Kimmie has been with AstraZeneca for over 20 years supporting early and late development programs across oncology, respiratory and infectious disease therapy areas and has experience and publications utilizing a wide range of methodologies including observational, qualitative, and health economic research. She holds a master of science degree in epidemiology from the University of Illinois Champaign-Urbana.
Vanessa Merker, PhD
Massachusetts General Hospital, Boston, MA, USA
Dr. Merker is a health services researcher at Massachusetts General Hospital and Assistant Professor in Neurology at Harvard Medical School. She specializes in qualitative and mixed methods, and has 13 years of experience in patient-centered research for rare diseases in the academic setting. As part of drug development efforts for neurofibromatosis and schwannomatosis, she leads patient interviews for three multisite clinical trials and organized a Critical Path Innovation Meeting with the FDA to discuss the role of embedded qualitative research within clinical trials for these diseases.
Eduard Sidelnikov, MD, PhD
Amgen (Europe) GmbH, Rotkreuz, ZG, Switzerland
Eduard Sidelnikov leads a Global HEOR Cardio, Bone, Neuroscience team at Amgen. His team provides full HEOR support including real world evidence generation, economic modeling and HTA deliverables. A graduate of Emory University and Universitat Pompeu Fabra Eduard has more than 25 years of experience epidemiology and health economics serving in various scientific and leadership positions in academia and pharmaceutical industry.
An Evidence Analysis Framework for Digital Health
Digital Conference Pass
Level: Intermediate
PURPOSE: With the growing availability of digital health interventions there is an increasing need for a structure to guide health economic and outcomes research (HEOR) and health technology assessments (HTA) by defining these technologies. In this workshop, the audience will learn about a proposed descriptive framework for defining digital health interventions meaningfully.
DESCRIPTION: A dedicated framework, the PICOTS-ComTeC, has been developed to overcome the issue that many terms which are currently used for digital health interventions are not sufficiently precise to allow for HEOR according to best practices. The ISPOR Digital Health interest group has done research on the current use of digital health terms and based on the shortcomings that were identified, a Delphi panel process was used to gain consensus on the most relevant domains to be specified in the definitions of digital health interventions for evidence summaries. The resulting framework called PICOTS-ComTeC is an extension of the well-established PICOT framework, which specifically addresses the variability of digital health interventions.
During the first half of the session, ZZ will introduce the research project and the challenges with digital health terms that were identified through the work of the interest group, RdB will present the PICOTS-ComTeC approach and case examples will be introduced by AB. The second half of the session will be interactive. The audience, including all people with an interest in HEOR and HTA related to Digital Health, will be guided by APH through an interactive exercise in which they apply the new framework to describe the digital health case examples. Finally, the audience and panel will share and discuss their experiences with the PICOTS-ComTeC.
Discussion Leaders
Zsombor Zrubka, MD, MBA, PhD
Óbuda University, Budapest, PE, Hungary
Zsombor is an associate professor and the head of the Health Economics Research Center at the University Research and Innovation Center at Óbuda University, Budapest, Hungary
Discussants
Artem Boltyenkov, PhD, MBA
Siemens Healthcare GmbH, Hellertown, PA, USA
Artem is a global head of Health Economics and Outcomes Research (HEOR) at Siemens Healthineers Diagnostics. He has an extensive background in HEOR in medical devices and diagnostics. He has more than 18 years of experience working in various HEOR roles for Siemens Healthineers in Germany and in the United States.
Artem is an Adjunct Professor at Rutgers School of Public Health, Piscataway, NJ, USA and at Ukrainian Free University, Munich, Germany. Artem is a visiting scholar at Feinstein Institutes of Medical Research, Manhasset, NY, USA.
Artem is the author of multiple books and peer-reviewed publications in medical journals on HEOR topics. He earned his PhD in Health Economics from the HHL, Leipzig (Dr. rer. oec.) and UFU, Munich (Dr. oec. pol. habil.) Universities in Germany. In addition, he has an MBA from the NYU Stern School of Business, Master’s in Computer Science and a Bachelor’s in Applied Mathematics from Taras Shevchenko National University, Kiev, Ukraine.
Rossella Di Bidino, PhD
Graduate School of Health Economics and Management (ALTEMS), Roma, RM, Italy
She is the head of the Unit on HTA for A.I. of the Advanced School of Health Economics and Management (ALTEMS) and senior HTA Expert at Fondazione Policlinico Universitario Agostino Gemelli IRCCS in Rome (Italy). She works in HTA since 2005. She is co-chair of the Hospital-Based HTA Interest Subgroup of the HTAi and member of the Digital Health Special Interest Group of the International Society of ISPOR.
Anke-Peggy Holtorf, PhD, MBA
PCIG @ HTAi.org, Basel, Switzerland
Anke-Peggy is the founder of the Health Outcomes Strategies GmbH consulting agency (www.health-os.com) with focus on value of healthcare products and related policy decisions for the in- and off-patent sector, reimbursement strategies, outcomes research and health economics, health technology assessment processes, payer interactions, product / device / service synergies.
Anke-Peggy acted as evaluator at the EU-commission on multiple occasions and is faculty member at the University of Utah College of Pharmacy where she reads in International Health Policy. She is engaged in workgroups for Precision Medicines Value (ISPOR), for Digital Health (ISPOR) and for Patient and Citizen Involvement (PCI) in HTA (as project coordinator for PCIG at HTAi) and leads projects on PCI in Low-and Middle-Income Countries and on PCI processes in HTA.
Real World Issues With Real World Data When Balancing Populations of Cancer Patients Who Have Histories, Treatment Sequences, and Biomarkers
Digital Conference Pass
Level: Intermediate
PURPOSE:
The utility of comparative-effectiveness evidence can be called in to question when real world data (RWD) cohorts differ in their treatment sequences before and/or after the index line of interest. Panellists will discuss methods available for balancing which can assist in preparing submissions for regulatory and HTA decision-making, providing case studies to illustrate the issues.
DESCRIPTION:
Dr. Adamson will open with an audience polling question to assess the variability in audience opinion of the optimal study designs to overcome challenges in balancing real world populations when treatment sequences and biomarkers differ. Dr Patel will share a recent case study in CAR-T and discuss the challenges of using RWD to support the launch. He will highlight the technical solutions that were difficult to implement and computationally expensive, but critical in the acceptance of RWD i.e., multiple imputation combined with propensity scoring, alongside the conduct of a simulation study to justify the choice of methods. Dr Vioix will talk about how differences in subsequent lines of therapy can confound comparisons (and thus acceptability to payers), demonstrated in a non-small-cell lung carcinoma (NSCLC) case study. The issues relating to the use of RWD in a biomarker defined population will also be highlighted as adding difficulty to cross-dataset comparisons. Dr Hatswell brings a focus on methodology, highlighting practical obstacles to the use of RWD – but how these may be (to an extent) be overcome to generate clinically meaningful evidence . These include data access issues, incompatible data classifications, and the uncertain place of indirect comparisons in the research pathway. His focus will be on providing the best possible estimates to inform healthcare decisions. Dr Adamson will transition the talks in to a discussion by challenging assumptions in the case studies (leaning on her experience with Flatiron) - and inviting audience participation.
Discussion Leaders
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is an epidemiologist and economist accelerating the development of valuable medicines and health policy. She serves as Principal Scientist at Flatiron Health in New York. Dr. Adamson advances artificial intelligence applications of natural language processing and machine learning in the curation of electronic health records for real world evidence with a team of engineers, scientists, and clinicians. She designs treatment comparative-effectiveness studies, codes mathematical models of disease, and delivers research for global health technology assessment. Dr. Adamson served as a former member of the White House COVID Task Force on Healthcare Resilience while lead pandemic data scientist in the West Wing. She is an Affiliate Professor at the University of Washington in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute. Dr. Adamson has held roles at the Bill and Melinda Gates Foundation Institute for Disease Modeling, Infectious Economics, the NIAID HIV Vaccine Trials Network, and Fred Hutch Cancer Center. She continues to serve as a leader in the International Society for Health Economics and Outcomes Research (ISPOR). Dr. Adamson holds degrees in pharmaceutical economics, public health epidemiology, and microbiology. She enjoys writing about the economics of infectious diseases at www.blytheadamson.com.
Discussants
Anthony Hatswell, PhD
Delta Hat, Nottingham, DBY, United Kingdom
Anthony Hatswell is a statistician & health economist. After his education at the University of York he spent time in pharma before moving to consulting where he specializes in applied statistics. His current research looks at cross study comparisons, and analysis of utility data.
Anik Patel, PhD
Kite Pharma, A Gilead Company, Santa Monica, CA, USA
Anik Patel is a pharmacist and health economist that has worked in clinical practice, academic and biotech roles. He is currently a director of Health Economics and Outcomes research at Kite, a leading CAR-T developer and manufacturer. His research focus is on comparative effectiveness, cost-effectiveness and real-world data analyses to support the value demonstration of cutting edge CAR-T programs. He has worked in a number of years in the biotech sector evaluating novel theraputics in oncology, ophthalmology and HIV. Prior to that, he worked in an academic setting studying infectious disease modeling with a particular focus on resource limited settings. He has graduate degrees from the University of British Columbia and University of Washington as well as Canadian and US clinical practice experience.
Helene Vioix, PharmD, MSc
Merck Healthcare KGaA, Darmstadt, HE, Germany
Pricing Pharmaceuticals for the Common Good: Is Value-Based Pricing Still the Right Solution?
Digital Conference Pass
Level: Introductory
ISSUE: Pricing pharmaceuticals for the common good: is Value-Based Pricing still the right solution?
OVERVIEW: Pharmaceutical innovation has contributed significantly to increasing life expectancy and quality of life globally; eased pressures on healthcare systems; and provided a range of broader benefits including contributing to improved workforce productivity and economic growth. Yet the pace and cost of breakthrough innovation are raising concerns around health system affordability and access challenges. Stakeholders are searching for a triple win that delivers for the common good by meeting the needs of patients, payers, and innovators both today and in the future.
Many countries claim to support value-based pricing whilst adopting policies (e.g. external reference pricing) that undermine it. Some advocates propose solutions including price transparency and cost-plus pricing claiming that they will lead to lower prices and greater access without noticeable impacts on the flow of innovation. Little attention is put to the demand side, and the needs of constrained healthcare systems. In this panel we ask whether value-based pricing and the current industry business model is still fit for purpose, by examining multiple perspectives: Thomas Allvin will present a recent conceptual framework - Equity-Based Tiered Pricing - as an evolution of VBP designed to ensure that ability to pay across countries is considered in the prices of innovative medicines, s, to reduce unavailability of new medicines and access delays. Oriol Sola-Morales will discuss VBP pricing approaches that can help meet the needs of all stakeholders in Europe. In light of the EU HTA regulation, he will discuss challenges in implementing a European strategy for value measurement to support an EU VBP pricing approach, and advocate for Value-Based HTA as a way forward. Eleanor Bell will discuss how far competing pricing models deliver for the common good based on economic theory, empirical scientific literature, and recent OHE research.
Moderators
Mikel Berdud, PhD
Office of Health Economics, London, LON, United Kingdom
Dr Mikel Berdud is Senior Principal Economist at the Office of Health Economics in the UK. His current research interests are the economics of innovation; including incentives for medical R&D, regulation of pharmaceutical markets, social welfare analysis, and competition. His recent research have focused on studying the implications of the value-based pricing using cost-effectiveness thresholds in social welfare and innovation, analysing the life-cycle value of medicines, innovative pricing and funding models for medicines, the economics of orphan drugs and AMR and R&D cost estimation methods.
Panelists
Thomas Allvin, MA
European Federation of Pharmaceutical Industries and Associations, BRUSSELS, VBR, Belgium
Thomas Allvin is Executive Director Strategy and Healthcare Systems at EFPIA. Before joining EFPIA in 2015, Thomas worked for the Swedish Ministry of Health, including as health attache at the Permanent Representation to the EU.
Eleanor Bell, MSc Health Economics and Decision Science
Office of Health Economics, London, LON, United Kingdom
Eleanor is a Senior Economist at the Office of Health Economics, where she has worked extensively on the value assessment and economics of innovation for vaccines and other complex technologies including digital health therapeutics.
Oriol Solà-Morales, MD, PhD
The HiTT Foundation, Barcelona, Spain
Oriol Solà-Morales, MD, Chair at Fundació HiTT, Barcelona, Spain, osola@fhitt.org
Dr. Solà-Morales is an internist who has led a regional HTA agency in Spain, with national and EU involvement. He has served in strategic innovation purchasing in two teaching hHospitals, has been an advisor to two Regional Ministers of Health, and has served on governmental research boards. He is currently affiliated with Universitat Internacional de Catalunya, where he teaches pharmacology and health economics.
Use of Joint Models in Health Technology Assessment (HTA) – The Future?
Digital Conference Pass
Level: Introductory
ISSUE:
Joint modelling [JM] has received considerable attention in the clinical and methodological research literature. The ability to model one or more time-to-event outcomes simultaneously with one or more longitudinal biomarkers offers potential benefits. Not only can the strength of association between biomarker and clinical event be assessed, but when one of the outcomes is death, the biomarker profiles are automatically adjusted for informative dropout. In addition, individual-level predictions of time to an event, conditional upon a biomarker profile up to a specific time point, can be obtained. A number of packages in R and Stata have been developed to implement joint modelling, and for more complicated applications a Bayesian approach using Markov Chain Monte Carlo (MCMC) methods can be adopted. However, there has been less development of methods to assess covariate selection and model performance, and to date applications of JMs in a HTA context have been limited. This Issue Panel will Illustrate their use with motivating examples, and discuss their benefits and limitations.
OVERVIEW:
Manuela Joore will introduce and provide an overview of Joint Modelling [JM] (10 minutes – including audience participation via
Slido ), and the three panellists will then introduce three potential application areas (in CVD, cancer & neurology) of JMs in HTA (10 minutes each) – Keith Abrams, adjusting longitudinal Quality of Life profiles for both informative dropout due to death and intermittent missing data in a CVD clinical trial; Louise Linsell, extrapolation of overall survival utilising longitudinal biomarker profiles in three phase I/II trials of adults and children with
TRK fusion cancer , and Varun Aggarwal, modelling the natural history of disease of patients with Duchenne Muscular Dystrophy. Manuela Joore will then moderate a discussion involving the audience and panellists on benefits and limitations of using JMs in HTA , before eliciting the audience’s final views (via
Slido ).
Moderators
Bram Ramaekers, PhD
Maastricht University Medical Centre+, Maastricht, LI, Netherlands
Panelists
Keith Abrams, PhD MSc CStat
University of Warwick and University of York, Coventry / York, Warwickshire / North Yorkshire, United Kingdom
Grace Lee, PhD
Critical Path Institute, Tucson, AZ, USA
Louise Linsell, DPhil MSc BSc CStat
Visible Analytics, Oxford, OXF, United Kingdom
Dr Linsell is a Principal Statistician at Visible Analytics Limited, a HTA consultancy company. She has extensive experience in medical statistics, clinical trials and epidemiology and was previously an Associate Professor of Biostatistics at the University of Oxford.
The Inherent Imprecision in Precision Medicine: How Do We Deal With the Resulting Uncertainties?
Digital Conference Pass
Level: Intermediate
ISSUE: Described as 'The right treatment to the right patient at the right time', precision medicine is conceptually deceivingly simple but challenges the way we generate, evaluate and act upon evidence as broad implementation becomes the pre-requisite rather than the consequence of successful evidence-generation. With its reliance on molecular diagnostics to identify the appropriate treatment, precision medicine represents an interesting challenge for determining the value at different points in its lifecycle due to interdependencies. This panel will look at the multiple sources of uncertainty and discuss pragmatic mitigation strategies. With the upcoming EU partnership for Personalised Medicine, the topic is very timely.
OVERVIEW: 10' BR will provide a patient perspective on PCM, provide relevant European context and introduce the session. 15' KT will report on the current phenomenon of national DRUP-like clinical trials, DLCTs, as pragmatic European multi-stakeholder precision medicine implementation initiatives, partially including pragmatic outcome-based risk sharing agreements between payers and manufacturers, followed by an HTA assessment once sufficient evidence has accumulated. 15' WO will introduce the HTAi-DIA working group's uncertainty framework for drug regulatory and HTA decision making and reflect on how to apply it to the context of a real-world precision medicine implementation initiative such as the DLCTs. 10' Panel discussion reflecting on the different types of uncertainties that arise for different stakeholder groups in precision medicine, their impact on the respective as well as other stakeholder group(s)- biological tumour heterogeneity e.g. limits the true validity of any diagnostic analysis - as well as potential mitigation strategies. 10' Q&A This session is relevant for anyone with interest in precision medicine: HTA bodies, payers, regulators, HEOR specialists, patient advocates and clinicians, data scientists.
Moderators
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
Dr. Ryll holds a medical degree from the Free University of Berlin, Germany, and a PhD in Biomedical Sciences from University College London, UK. After losing her husband to Melanoma, she founded the Melanoma Patient Network Europe and developed a special interest in patient-centric clinical research and innovation, innovative trial designs and novel drug development concepts. Between 2019 and 2021, she was member of the first EU Cancer Mission Board where her particular interest has been how to best leverage the potential of personalized medicine for patients and society through novel forms of collaboration and the support of health policy and governance. Currently, she works as strategist at SIR, Stockholm School of Economics' Research Institute for Mission Zero Cancer, an innovation ecosystem in health financed by Vinnova, Sweden's innovation agency.
Panelists
Inka Heikkinen, MSc, MBA
MSD, Copenhagen, 84, Denmark
Lead of the EFPIA Joint Scientific Consultation workstream. Special focus on regulator-EMA interaction, participated in various discussions and research activities (eg. on unmet medical need). Active in EHDS and RWE discussions and advocacy within EFPIA. Academic degrees on health economics and in policy, an Executive MBA degree with specialisation in finance.
Kjetil Tasken, MD/ PhD
Oslo University Hospital, University of Oslo, Oslo , Norway
Professor Kjetil Taskén has been key in building Norway's national cancer precision medicine initiative and now coordinates the Cancer Mission PRIME-ROSE project for DRUP-like clinical trials across Europe. He authors over 300 publications and inventories>20 patents (h-index =65). He won the King Olav V’s Prize for Cancer Research in 2016 and the University of Oslo Innovation Prize in 2023 and is Vice-President of the Norwegian Academy of Science and Letters. Current research is in tumor immune evasion mechanisms and functional precision medicine for different solid and blood cancers.
8:30 - 9:30
Breakfast Break With Exhibitors
Located in the Exhibit Hall
8:30 - 11:30
Exhibit Hall Open
8:45 - 9:45
Podium Sessions
Differences and New Developments in HTA Around the World
Digital Conference Pass
This podium session will compare the technology appraisal approaches of HTA agencies. A special emphasis will be made on changes in NICE's appraisal process.
Moderator
Mehtap Tatar, PhD
Vitale Health Economics and Policy, London, United Kingdom
Prof. Dr. Mehtap Tatar is a health economics and health policy expert. She received her postgraduate degrees from the London School of Economics and Nottingham University. She is the founder and director of two consulting companies based in Turkey (Polar Health Economics and Policy -www.polarsaglik.com) and in London (Vitale Health Economics and Policy-www.vitalehealtheconomics.co.uk). Her main areas of expertise are economic evaluation, pricing and reimbursement of pharmaceuticals and medical devices, health technology assessment and health financing.
P46: Drivers of Health Technology Assessment Methods and Process Reforms: An International Comparison of Real World Evidence, Patient Involvement and Discounting
9:00AM - 9:15AM
Bell E1 , Cubi-Molla P1 , Garau M2 , Kumar G2 , Pan J3 , Radu P2 , Baxter C4 , Gilardino R 5 , Goodall M6 , Nelson K7 , van Bavel J8 1 Office of Health Economics, London, LON, UK, 2 Office of Health Economics, London, UK, 3 Adelphi Values PROVE, Bollington, Cheshire, UK, 4 Vertex Pharmaceuticals, London, London, UK, 5 MSD, Dubendorf, ZH, Switzerland, 6 Goodall HTA Consulting, Manchester, Manchester, UK, 7 Merck & Co., Inc., Philadelphia, PA, USA, 8 MSD, Sydney, NSW, Australia
OBJECTIVES: Our research aims to understand what drives change in Health Technology Assessment (HTA) agencies’ methods and processes (M&P), specifically positions on real-world evidence (RWE), patient involvement in HTA, and discounting, in Australia, Belgium, Canada, Denmark, England, France, Germany, Italy, Portugal, Sweden, Singapore, Spain, Taiwan, and the Netherlands.
METHODS: We conducted a pragmatic review of grey and academic literature to identify relevant summaries in HTA M&P and HTA agency documentation relating to the topics of interest over the last 20 years. We interviewed 29 academic and HTA experts to understand motivations and constraints for M&P reforms, and influence on or by other HTA agencies.
RESULTS: HTA agencies’ stances on the topics of interest lie on a spectrum and have evolved over time. We mapped the timeline of M&P changes and identified drivers acting as enablers or constraints to reforms, such as: stakeholders; country-specific context (healthcare policy, legal and political factors); and cross-border context (e.g., other countries’ HTA practices, external shocks).
All HTA agencies consider RWE to an extent in decision-making, with recent formalised processes created by NICE, CADTH and RedETS. NICE, IQWiG and CADTH have clearly defined opportunities for patient consultation, such as feedback on recommendation. In contrast, other agencies do not explicitly state whether they consider patient evidence. Base-case discount rates have remained mostly unchanged over time, and all agencies have permitted sensitivity analyses on rates for historical or international comparison.
CONCLUSIONS: Our work provides an evidence-based overview of the drivers of HTA M&P reforms internationally. While some HTA agencies have formalised or evolved their positions on RWE, patient involvement and discounting (IQWiG, NICE and CADTH were first-movers, respectively), others lack an explicit stance. HTA agencies themselves and national contextual factors have mainly driven reform. However, international collaborations have potential to accelerate M&P reforms and help resolve HTA challenges.
P45: Are There Any Differences in the Recommendations for Ultra Orphan Medications: An Assessment of NICE, GBA, and HAS
8:45AM - 9:00AM
Bhatnagar T1 , Siroula M1 , Gupta J 2 , Siddiqui MK1 , Nielsen SK3 1 EBM Health Consultants, New Delhi, DL, India, 2 EBM Health Consultants, New Delhi, DL, India, 3 V2A Consulting GmBH, Lausanne, Vaud, Switzerland
OBJECTIVES: Ultra orphan drugs pose challenges in the health technology assessment (HTA), reimbursement, and pricing due to limited patient population and data, resulting in high decision uncertainty. Considering these challenges, we aim to examine the outcomes for ultra orphan drugs appraised by NICE, GBA, and HAS.
METHODS: All highly specialised technologies (HST) appraisals submitted to NICE between 2015 to 2023 were reviewed. The identified interventions were further searched on the GBA and HAS websites. Final recommendations along with the justification and categories for additional clinical value (Major, Considerable, Minor, Non-quantifiable, No benefit, and Less benefit for GBA and Major, Substantial, Moderate, Minor, and No improvement for HAS) were recorded. Only dossiers available in English language were considered.
RESULTS: Out of the 22 drugs appraised by NICE, 64% were fully recommended (i.e., in line with their marketing authorisation), and 36% were recommended with restrictions. All recommendations included a simple discount-based patient access scheme. There were 13 matched pairs for NICE versus GBA and of these, 23% were given Considerable/Major rating by GBA, while 62% had Non-quantifiable additional clinical benefits. Similarly, for the 18 drugs assessed by both NICE and HAS, clinical benefit was rated by HAS as Minor improvement/No improvement in 44% and as Substantial improvement in 22% submissions. Of the 10 matched pairs for all three agencies, 60% were rated Non-quantifiable by GBA while the same was considered as Minor (30%), Moderate (20%) and Substantial improvement (10%) by HAS. Overall, high uncertainty regarding the clinical effectiveness of the drugs was observed across the three agencies.
CONCLUSIONS: Despite similar clinical data, there is inconsistency in recommendations across different HTA bodies which can impact the pricing and reimbursement decisions. A Joint clinical assessment can potentially harmonise these inconsistencies and ensure equal access to innovative treatments for patients with ultra orphan diseases across Europe.
P48: New NICE Proportionate Approaches: Are They Any Faster or More Successful?
9:30AM - 9:45AM
Andrews R 1 , Hall D2 , Macaulay R3 1 Precision Advisors, London, UK, 2 Precision Advisors, London, Greater London, UK, 3 Precision Advisors, Edinburgh, UK
OBJECTIVES: The National Institute for Health and Care Excellence (NICE) assesses the incremental clinical- and cost-effectiveness of healthcare technologies to inform public reimbursement recommendations in England and Wales. In 2022, NICE implemented a pilot for a new proportionate approach to technology appraisals, aiming to apply faster evaluation processes to simpler, low-risk treatments. This research assesses the impact of this approach on timing and outcomes of NICE recommendations.
METHODS: All NICE proportionate approach appraisals were identified (01-JAN-2022–17-JUL-2023) from https://www.nice.org.uk/ . Parameters were then compared to NICE Single (STA) and Multiple Technology Appraisals (MTA) taking place in 2021/2022 (21-APR-2021–30-MAR-2022) from https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance/data/appraisal-recommendations ).
RESULTS: Five products have undergone NICE appraisals under the proportionate approach (somatrogon, vutrisiran, nintedanib, eptinezumab, and nivolumab), with four more currently being assessed. Four of the pharmaceutical products were recommended and one was optimised (restricted), with an average time between ‘invitation to participate’ and publication of 34 weeks (range: 22-40 weeks). In the 2021/2022 period, 104 NICE HTA recommendations for pharmaceutical products were identified. Overall, 71% of outcomes were positive (recommended, optimised, or approved via Cancer Drug Fund), 2% were only in research, 9% were not recommended, and 19% were non-submissions. The average appraisal time was 73 weeks (IQR: 50-90 weeks) for all recommended 2021/2022 appraisals.
CONCLUSIONS: Our analysis suggests that NICE’s proportionate approaches accelerate the assessment of products that meet the eligibility criteria by an average of 39 weeks. However, it is important to note that these pilot assessments were intended to test the viability of this approach and the products assessed are likely to have been carefully selected. It will be interesting to monitor outcomes from subsequent use of NICE’s proportional approaches to see the extent to which they are adopted more widely and to which they shorten assessments.
P47: How Do HTA Agencies Manage Uncertainties in the Long-Term Clinical Benefit of Gene Therapies? An Analysis of Recent Gene Therapy HTAs in England, Germany, France, Italy, Canada, and the United States
9:15AM - 9:30AM
McCormick B1 , Sheppard C1 , Hutt E1 , Schmitz S1 , Kumar A 2 1 Partners4Access, London, UK, 2 Partners4Access, Enfield, UK
OBJECTIVES: To date, seven gene therapies (GTs) have European regulatory approval and have been reimbursed at significant upfront costs. Assessing the long-term benefits of one-time GTs is challenging for payers due to the short duration of clinical data at the time of Health Technology Assessment (HTA). This research aims to understand the methods used by HTA agencies to assess the durability of GTs and whether the concept of biological plausibility is considered by payers.
METHODS: The methods manuals of six HTA agencies (NICE, G-BA, HAS, AIFA, CADTH, and ICER) were examined to compare guidance on the procedures used to estimate long-term durability. The appraisals of three GTs (voretigene neparvovec, onasemnogene abeparvovec, and valoctocogene roxaparvovec) were also analysed.
RESULTS: There is limited explicit guidance in HTA method manuals on how agencies determine long-term benefit in light of short-term clinical evidence, beyond the traditional economic modelling methods used in select countries. GT-specific guidance was also lacking. Only the NICE methods manual explicitly stated that clinical expert opinion could inform whether it was biologically plausible that the clinical effect would be maintained. Analysis of three GTs revealed that clinical expert opinion and biological plausibility were utilised to varying degrees between HTA agencies, with NICE, CADTH and AIFA employing biological plausibility. Four agencies required real-world evidence or innovative access agreements (IAAs) to help mitigate the financial risk of reimbursing GTs for which the durability of effect is uncertain.
CONCLUSIONS: Most agencies use traditional economic modelling or post-HTA evidence generation mitigation strategies (e.g., IAAs) to manage uncertainty in long-term benefit of GTs. IAAs may be practically unsustainable with the expected influx of GTs in coming years, due to the burden associated with implementation. HTA agencies, manufacturers, and the scientific community must adopt novel methods to suitably assess the long-term benefit of GTs during HTA.
9:00 - 11:30
Poster Session 5
Live
9:15 - 9:45
Exhibit Hall Theater
Art of the Possible - Creating Value With End-to-End Market Access Strategies
Digital Conference Pass
It is never too early to begin planning your market access strategy. In our session, we will explore the dynamics and complexities of bringing innovative medicines to market providing insights on the value of creating end-to-end market access strategies for a successful product launch.
Sponsored by WEP Clinical
Sponsor
WEP Clinical
Speaker
Penny Fairmann, BSc (Hons)
WEP Clinical, London, United Kingdom
Penny is the Associate Director Market Access at WEP Clinical, supporting clients to navigate the complexities of EU pricing and reimbursement landscape.
Penny has been a successful market access specialist in the Biopharmaceutical industry for 20 years having extensive experience in design and delivery of value materials to support multiple specialised medicine launches. Penny has held a number of infield and senior strategic market access management roles across small and medium biopharmaceutical companies in the UK including UCB and Jazz Pharmaceuticals.
Heather Masters, BSc (Hons)
WEP Clinical, London, United Kingdom
Heather is responsible for Market Access at WEP Clinical, working with our partners to secure optimal pricing, access and reimbursement of licenced portfolios. Heather has been a successful business leader in the Pharmaceutical industry for nearly 20 years, and has extensive knowledge of global healthcare systems and expertise in developing Market Access strategies for innovative therapies in Oncology and Rare Diseases. Heather has held a number of senior management roles in Commercial Marketing & Market Access for Novartis AG and has worked as a Business Consultant with various biopharmaceutical companies across a wide range of therapy areas.
10:00 - 11:00
Breakouts: IP, WS, & OBS
Quality Over Quantity: How Do Clinical Trial Sponsors Balance the Evidence Needs of Regulatory and Reimbursement Bodies When Assessing Patient-Reported Outcomes (PRO) in Oncology Trials?
Digital Conference Pass
Level: Intermediate
ISSUE: Assessing the health-related quality of life (HRQoL) of treated cancer patients remains paramount for sponsors, regulators, reimbursement bodies, clinicians and patients. Patients are the best source of data on the frequency and severity of disease-related symptoms, treatment side effects, and the impact of treatment on functioning and well-being; measuring these issues in a way that minimizes patient burden and limits missing data is paramount. Doing this in a way that satisfies all stakeholders can be challenging, as there appear to be differing evidence requirements among review bodies, leading to alternative approaches of using full generic and disease-specific PRO questionnaires vs. targeted use of specific scales and items. These contrasting perspectives will be discussed in this plenary session.
OVERVIEW: Oncology clinical trials have historically collected PRO data through standard generic HRQoL questionnaires and disease-specific modules. Treatment paradigms have evolved since these instruments were developed, meaning current clinical trials may include questions that have little or no relevance, while missing important disease or treatment-related concepts.
FDA’s patient-focused drug development (PFDD#3) guidance describes the importance of starting with a clear understanding of the disease or condition in order to select, modify or develop validated PRO measures. The outcomes measurement community (including the EORTC), based on feedback from patients, is moving towards assessments that are tailored, through the use of modular item libraries, to maximize relevance to the person's disease and treatments. In contrast, some reimbursement bodies require complete validated, standardized generic and disease-specific questionnaires. This plenary discussion aims to: 1) Clarify positions on the requirements for oncology clinical trial PRO evidence across these critical groups in drug development 2) Identify areas of agreement among them 3) Brainstorm on actions that can be taken to bridge the remaining gaps. Presentation time totals 40 minutes with 20 minutes for audience questions and discussion.
Moderators
Peter Trask, PhD
Genentech, South San Francisco, CA, USA
Moderator, Panel on PROs in early clinical trials, Friends of Cancer Research 2022 Annual Meeting.
Over 80 publications on PROs with the majority being on outcomes in oncology patients.
Panelists
Vishal Bhatnagar, MD
Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, MD, USA
Vishal Bhatnagar, MD, is a medical oncologist/hematologist and the Associate Director for Patient Outcomes in the OCE. His interests include patient reported outcomes, patient preference and incorporation of patient experience in oncology trials. His work focuses on the operational management of the OCE’s Patient-Focused Drug Development program. Additionally, Dr. Bhatnagar has a strong clinical interest in multiple myeloma and has previously served as an Office of Hematology and Oncology multiple myeloma scientific liaison. Dr. Bhatnagar received his BA in Political Science and his medical degree at the George Washington University. He completed his internal medicine residency and hematology/oncology fellowship at the University of Maryland.
Kirsten Janke, PhD
Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), Cologne, Germany
Dr. Kirsten Janke is a Research Associate with supervisory responsibilities in the Drug Assessment Department of the Institute for Quality and Efficiency in Health Care (IQWiG), Germany’s Health Technology Assessment body. In this role she is responsible for supervising drug assessment procedures and for the development of methods and processes for these assessments. Dr. Janke holds a Diploma In Biology from the Heinrich Heine University Düsseldorf and a Dr.rer.nat. from the University of Duisburg-Essen
Claire Piccinin, MSc.
European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium
Claire has been a researcher in the EORTC’s Quality of Life Department since 2017. She oversees the use of the EORTC Item Library alongside the Item Library Support Team, providing recommendations on the implementation of flexible Item Library-derived measures in PRO assessment strategies. Her research is focused on evaluating trends in EORTC Item Library usage, implementing standardized frameworks to support item/measure selection, and developing guidelines and recommendations to facilitate the use of item libraries for PRO measurement in cancer clinical trials and as part of routine care.
How Do We Attain Sustainability, Affordability and Availability of CAR T Therapies?
Digital Conference Pass
Level: Intermediate
ISSUE: As novel treatments with high therapeutic potential, chimeric antigen receptor T-cell ( CAR T) therapies represent a significant advancement in medical innovation. At the same time, pricing and reimbursement (P&R) in Europe is particularly challenging for CAR Ts because of their unique characteristics and high price. To ensure patients can benefit from these treatments, there is a need to better understand the different challenges faced by healthcare systems, payers and manufacturers, and what ways forward might look like for all stakeholders involved. Determining how CAR Ts can be sustainable, affordable and available thus requires exploring their value and challenges, and finding suitable solutions for managing evidence and/or affordability concerns while bringing these valuable therapies to patients.
OVERVIEW: An overview of CAR Ts will be presented, including how they differ from more conventional therapies, the current challenges they face, and potential solutions (10 minutes, moderator). The core themes around the presented challenges and possible solutions for CAR T innovation will first be addressed from the policy/payer perspective, including whether the challenges are comprehensive and accurate, and whether the solutions are feasible from the point of view being represented (13 minutes, Sarah Garner, WHO). These core themes will then be scrutinised and addressed in the same way from the perspective of CAR T development and manufacturing (13 minutes, Anne Kerber, BMS), and from the perspective of CAR T economics (13 minutes, Konstantinos Lykopoulos, Kite).
The conversation will then be opened for the audience to comment and ask questions to the panel. The intention of the discussion will be to explore whether there is any common agreement among the different perspectives, and what potential ways forward could look like to make CAR T innovation sustainable for all stakeholders (11 minutes). This panel session may benefit manufacturers, payers, policy makers and patient organisations.
Moderators
Amanda Whittal, PhD. Health Psychology
Dolon Ltd, London, LON, United Kingdom
Amanda Whittal is a health psychologist with a combined research and consultancy background in health policy for rare and innovative medicines. She has been involved in developing and publishing policy recommendations for orphan medicines through the Horizon 2020 project 'IMPACT-HTA', has published on the economics of gene therapy, and has developed guidance frameworks for ATMPs and managed entry agreements.
Panelists
Anne Kerber, MD., Degree in Pharmaceutical Medicine
Bristol-Myers Squibb, Nieder Olm, Germany
Anne Kerber serves as senior vice president, head of Cell Therapy Development at Bristol Myers Squibb and leads all clinical trial and development activities of BMS’ cell therapy portfolio. Anne is an experienced leader and drug developer, with particular expertise in cell therapies. Prior to BMS, she worked at Kite, a Gilead Company, as vice president, Clinical Development. Before that, she held several leadership roles at Affimed.
Anne received her M.D. from Philipps University in Marburg, Germany and completed her clinical training in Internal Medicine at Johann Wolfgang Goethe University Hospital in Frankfurt. She also holds a degree in Pharmaceutical Medicine from the University of Basel, Switzerland.
Konstantinos Lykopoulos, MSc. International Health Policy and Health Economics
Kite, A Gilead Company, London, United Kingdom
Konstantinos Lykopoulos will discuss challenges and solutions to CAR T sustainability, affordability and availability based on his extensive experience in CAR T value and access. His nearly 15+ years of experience in oncology and market access enables him to look particularly at the unique economics of CAR T development, manufacturing and delivery to address the key challenges he sees for CAR T P&R in Europe, and why he feels the proposed solutions are feasible or not.
Tarang Sharma, MS, MPH, PhD
WHO Regional Office for Europe, Copenhagen, 84, Denmark
Tarang Sharma has 20+ years of post-graduate experience as a biologist, epidemiologist and health economist. She has worked with regulatory submissions, health technology assessments and clinical guidelines, as an author, methodologist, reviewer, editor and decision-maker. Tarang Sharma has until recently worked for the Agenda, Policy & Strategy unit in the Immunization, Vaccines and Biologicals department at the World Health Organization HQ in Switzerland, supporting evidence to policy recommendations for new COVID-19 vaccines as well as cross-cutting considerations by the Strategic Advisory Group of Experts on Immunization (SAGE) and has previously worked for evidence to policy impact and health research mechanisms both at WHO HQ and their WHO European Regional Office (WHO Europe) in Denmark as a senior consultant and a technical officer. She now acts as the HTA focal point and is the Technical Officer for the Novel Medicines Platform at WHO Europe.
She has also previously worked for three different national governments: in USA (American Museum of Natural History) and in Europe for two HTA agencies: the National Institute for Health and Care Excellence [NICE] as a Senior Analyst in the United Kingdom and for the Danish Medicines Council: Medicinrådet, as a Specialist Consultant in Denmark. In UK, initially as a health economist at the University of Sheffield that hosts the NICE Decision Support Unit apart from undertaking individual HTAs for NICE, she was responsible for setting methods and standards for HTA. Then later, directly for NICE initially as a technical lead for the guidelines and then later with the science, policy, and research unit, as a senior analyst she managed a technical scientific team, to develop and improve methods, research, and partnerships for HTA. She also represented NICE at the international collaborations many funded by EU such as EUnetHTA, DECIDE apart from GRADE, social values in HTA etc. At Medicinrådet she helped establish the methods and processes for the use of quality adjusted life years approach as well as priority setting for HTA assessments, apart from conducting individual HTAs for Denmark.
She has also worked for non-governmental organizations (NGOs) with the Nordic Cochrane Centre (now referred to as Cochrane Denmark) in Denmark as well as part of the Cochrane Central Executive Team at their headquarters in London, UK. As a Research fellow, with Cochrane Denmark, she worked to improve transparency of clinical trials of medicines and reporting of adverse events and quality of life outcomes in collaboration with regulators EMA and the UK’s MHRA, for which she received a PhD from the University of Copenhagen. Later at the Cochrane headquarters, she established a new workstream to manage the editorial policies, process, budget and team for the Cochrane’s Fast-Track service (now the centralised service). She has also worked at the grass-roots level for NGOs in India for Akanksha and Kutumb foundations for nutrition and public health for populations living on the streets.
She has also worked for two pharmaceutical companies Novo Nordisk A/S in Denmark and Ranbaxy Laboratories Ltd (later bought by Sun Pharma) in India. At Novo Nordisk, she assessed quality of life and other patient reported outcomes from Phase III trials and conducted relevant observational studies for new medicines for submissions to authorities including discrete choice experiments, willingness to pay assessments, patient and carer surveys etc. As a Research Scientist at Ranbaxy, she conducted pre-clinical and Phase I trials for new medicines for their development as well as for generics.
She is an honorary associate editor for the Frontiers in Public Health: Health Economics and has previously been an editor for BMC journal Cost-effectiveness and Resource Allocation, BMJ, Cochrane and a series editor for the WHO Europe publication Health Evidence Network (HEN). She has a MS in biology (applied recombinant DNA technology, Jan 2003) from New York University, an MPH (specialised in health economics, November 2008) from University of Sheffield and MPH (July 2008) and a PhD in public health and epidemiology from University of Copenhagen (May 2018).
Disease-Specific Reference Models for Health Technology Assessment: Necessity or NICE-to-Have?
Digital Conference Pass
Level: Introductory
ISSUE: Many disease areas have numerous technologies as potential treatments, resulting in complex sequential and multi-comparator decision spaces. Depending on the reimbursement process, model development and evidence synthesis efficiencies may be possible by developing a disease-specific reference model. As the development and interest in disease-specific reference models become more ubiquitous, this panel offers the opinions of 3 stakeholders on the advantages and challenges they foresee with disease-specific reference models.
OVERVIEW: There is significant literature on ‘reference models’, which enable a single model to consider multiple decision nodes along a treatment pathway, informing multiple decision problems, minimising duplication of effort and improving internal consistency. However, their use for reimbursement has been relatively limited.
Many HTA agencies are seeing an increase in technologies needing appraising. Several agencies are developing reference models, creating an opportunity for innovative methods to help ensure the sustainability of HTA processes across established and newer agencies. Companies undertake significant work to develop and adapt cost-effectiveness models for reimbursement submissions in many counties. Disease-specific reference models would change the dynamic between the HTA agency and industry. Companies must consider the potential for a more efficient and speedy process against the challenge of ensuring the reference model best reflects a value proposition and accommodates the latest relevant evidence. The moderator will provide an overview of disease-specific reference models for 5 minutes to ensure all attendees understand the context of the session. Then each panellist will present the expected impact on their organisation. The proposed speakers have been specifically selected to ensure that diverse backgrounds and views are represented. To ensure the issue panel is interactive and provides a learning opportunity for attendees, live polling functionality will be utilised, and time will be reserved for a moderated Q&A session. This panel will be of interest to researchers, clinicians, HEOR industry colleagues and payers.
Moderators
Zoe Garrett, MRes, MPhil
National Institute for Health and Care Excellence (NICE), Manchester, United Kingdom
Zoe Garrett is a senior technical adviser in the Science Policy and Research programme and the National Institute for Health and Care Excellence (NICE). Zoe joined NICE in 2005 working in the Technology Appraisal programme and then in the NICE scientific advice programme. In 2012, Zoe started supporting NICE’s international HTA activities and managing the NICE-led work for the European Network for Health Technology Assessment (EUnetHTA) joint actions. Zoe supports NICE’s international partnerships, managing relationships with other HTA agencies and supporting NICE teams to collaborate.
Panelists
Eric Low, BA
Eric Low Consulting, Haddington, ELN, United Kingdom
Eric Low is the Founder of Eric Low Consulting, specialising in health technology assessment, market access, patient engagement, and life sciences and healthcare policy. Before this, Eric established and served for over 20 years as CEO of Myeloma UK. Eric is committed to improving patient outcomes and puts patients at the centre of everything he does. He holds several Board, honorary, advisory and voluntary positions and advises numerous medical, research and health-focused charities on a pro bono basis. He was awarded an OBE for services to charity in the Queen’s Birthday Honours 2012.
Oresta Piniazhko, PhD
State Expert Center of the Ministry of Health of Ukraine, Kyiv, Ukraine
Oresta Piniazhko, PhD, Director of HTA Department at State Expert Centre of Ministry of Health, Ukraine.
Oresta is an experienced expert in HTA, pharmaceutical policy and implementation practitioner. She holds a PhD degree in Pharmacoeconomics and is currently holding a position of Director of HTA Department at the State Expert Center of the Ministry of Health of Ukraine, ensuring management and implementation of the best international practices of HTA into health care system of Ukraine since February 2019. Being a dynamic communicator she is also a President of Ukraine ISPOR Chapter since 2017 and before ISPOR Ukraine Students Network (2015-2017). Oresta is visiting lecturer at The Institute of Business Education of Vadym Hetman Kyiv National Economics University and senior lecturer at Danylo Halytskyi Lviv National Medical University.
Jon Tosh, PhD, MsC
GSK, Middlesex, LON, United Kingdom
Dr Jon Tosh is a Director of UK Health Outcomes at GSK.
Jon has over 15 years' experience of health economics research, with over 25 peer-reviewed publications. Jon has contributed as an ISPOR Task Force member and short course instructor.
He was awarded an NIHR fellowship, resulting in a PhD where he applied disease modelling and optimisation methodologies in an economic evaluation of sequential therapies for rheumatoid arthritis.
Jon's expertise lies in cost-effectiveness modelling, simulation, optimisation, economic evaluation, and HTA. He has an MSc (London School of Economics) in International Health Policy and Health Economics, and a PhD (University of Sheffield) in Health Economics.
Point-of-Care Diagnostics for Antimicrobial Resistance. Will the Experience of the COVID-19 Pandemic Increase the Likelihood of Their Uptake?
Digital Conference Pass
Level: Introductory
ISSUE: The issue to be presented and debated is whether the accelerated uptake of diagnostics seen in the COVID-19 pandemic increases the likelihood of the uptake of other point-of-care (PoC) diagnostics, specifically for guiding appropriate antibiotic prescribing.
OVERVIEW: In 2019, 4.95 million deaths were associated with drug-resistant bacterial infections and 1·27 million deaths worldwide were directly attributed to antimicrobial resistance (AMR) making it a priority area for interventions. One potential approach for tackling AMR being explored by the Innovative Medicines Initiative-funded VALUE-DX project is using PoC diagnostics for community-acquired respiratory tract infections to guide antibiotic prescribing.
During the COVID-19 pandemic PoC diagnostics were introduced in a timely manner that was unprecedented for similar in-vitro diagnostics, due to the perceived urgency for their need by multiple stakeholders and the WHO’s emphasis on testing. Given the pressing need for implementation of effective measures to reduce AMR, will PoC diagnostics for AMR be adopted as widely as they were for COVID-19, if proven to be effective? What factors will affect their uptake, and will they vary by geographical region? What learnings from the COVID-19 pandemic should be considered by different stakeholders? What evidence will HTA bodies need to see and what are the perceived challenges in demonstrating the value of diagnostics for AMR? Dalia Dawoud will introduce the topic providing an overview of the extent and impact of AMR. Alec Morton will present key considerations when assessing the value of interventions for AMR, with a focus on diagnostics. Jean-Louis Tissier will provide the industry perspective regarding the barriers to uptake of PoC diagnostics and demonstrating their value. Fatima Salih will present key themes from meetings with an expert advisory panel convened for the IMI VALUE-Dx project. There will be three 10-minute presentations, followed by panel/audience discussion with polling questions administered to seek the audience’s views.
Moderators
Dalia Dawoud, PhD
National Institute for Health and Care Excellence, London, LON, United Kingdom
Dalia Dawoud, PhD, is Associate Director (Research) at the National Institute for Health and Care Excellence (NICE). She has practised as health economist and researcher for over 20 years. Her current work is focused on advancing HTA and clinical guideline development methods through research.
Dalia leads NICE newly established HTA Innovation Laboratory (HTA Lab) as well as NICE’s portfolio of European Commission funded projects, such as IMI EHDEN and HORIZON 2020 HTx projects, with cumulative funding of over 5 Million Euros. She is widely published in the area of health economics and outcomes research and serves as Associate Editor of ISPOR journal Value in Health and as Director on ISPOR Board of Directors (2023-2026).
Panelists
Alexander David Morton, PhD
National University of Singapore, Paisley, RFW, United Kingdom
Alec Morton is Professor and Head of Department of Management Science at Strathclyde Business School, University of Strathclyde in Glasgow, Scotland. He has degrees from the University of Manchester and the University of Strathclyde. He has worked for Singapore Airlines, the National University of Singapore, and the London School of Economics, has held visiting positions at Carnegie Mellon University in Pittsburgh, Aalto University in Helsinki, the University of Science and Technology of China (USTC) in Hefei, and the National Audit Office and is a member of the International Decision Support Initiative. His main interests are in decision analysis and health economics. His research is funded by the European Commission, the Department of Health, the Medical Research Council and Engineering and Physical Sciences Research Council, and the Chief Scientist's Office of the Scottish NHS.
Alec has been active in the INFORMS Decision Analysis Society, EURO and ISPOR. He is on the Editorial Board of Decision Analysis and is an Associate Editor for the EURO Journal on Decision Processes, the Transactions of the Institute of Industrial Engineers, and OR Spectrum. Past consulting clients include the National Audit Office, the Department of Health, the Environment Agency, the Nuclear Decommissioning Authority and the Global Fund to Fight AIDS, Tuberculosis & Malaria. His papers have won awards from the International Society for Pharmacoeconomics and Outcomes Research and the Society for Risk Analysis. His book Portfolio Decision Analysis with Jeff Keisler and Ahti Salo won the INFORMS Decision Analysis Society publication award in 2013 and his paper "CUT: A Multicriteria Approach for Concavifiable Preferences" (with Nikos Argyris and Jose Figueira) was a finalist for the same prize in 2016 .
Fatima Salih, MSc
National Institute of Health and Care Excellence, London, United Kingdom
Fatima Salih is a Scientific Adviser at the National Institute for Health and Care Excellence (NICE). She holds an MSc in Public Health/Health Economics from the London School of Hygiene and Tropical Medicine and has over 10 years experience in health economics and outcomes research gained in HTA organisations and private consultancy environments. She is currently the operational lead for NICE tasks on Innovative Medicines Initiative-funded projects thus has experience of undertaking research as part of public-private consortia. She has also worked in public health in government organisations both in the UK and in Sudan.
Jean-Louis Tissier, MSc
bioMérieux, Marcy l'Etoile, France
Jean-Louis is Vice President, Public and Government Affairs – AMR at bioMérieux since October 2019. As a world leader in in vitro diagnostics, focusing on infectious diseases, bioMérieux has a long-standing commitment to fighting AMR. This commitment is at the heart of the Company’s mission to serve public health all around the globe.
Jean-Louis joined the company in 1984 holding roles of increasing responsibility, most recently as Vice President Global Marketing in Microbiology (2007-2011) and Vice President Northern Europe (2011-2019) which allowed him to accumulate rich experience in the field of in vitro diagnostics. He graduated from the Business School of the Conservatoire National Des Arts et Métiers in Paris and received his Master degree in Biology from the University of Paris.
Podium Sessions
Medication Use and Its Consequences
Digital Conference Pass
Moderator
Steffen Wahler, MD; MA
St. Bernward GmbH, Hamburg, Germany
Steffen is Managing Owner of Hamburg-based St. Bernward Consulting. He has held leading positions in German health insurance, medical device and pharmaceutical industry. He served as director in the German pharma trade association (vfa) and was in the curator-board of IQWiG and the DIMDI-HTA division. Steffen obtained his doctorate in medicine at Rostock University and specialized as diabetologist. He earned degrees in economics at Universities of California and York/England as well as at INSEAD, France. He is regularly publishing in his and adjacent fields, like epidemiology and care organization research and co-authored the German Health Economics guidelines, the Hanover Consensus.
P56: Trends in Prescription Drug Use Among Adults With Diabetes in the United States From 1999-2018
10:45AM - 11:00AM
Li S 1 , Pan S2 , Jiang S3 , Lyu B4 1 School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, Jiangsu, China, 2 Guizhou Medical University, Guiyang, Guizhou, China, 3 Peking University, Beijing, China, 4 Peking University, Beijing, Beijing, China
OBJECTIVES: Diabetes care is associated with substantial medical and financial burden to individuals and society. Understanding the patterns of medication use can help inform clinical and pharmaceutical practice.
METHODS: To assess the trends in prescription drug use among diabetic patients from 1999 to 2018, we conducted a series of cross-sectional analysis from adults with diabetes in the United States participating in the National Health and Nutrition Examination Survey (NHANES), a nationally representative survey of the civilian noninstitutionalized population.
RESULTS: Among 8,895 adults with type 2 diabetes (T2D), 88.4% [87.3%-89.4%] reported using prescription medication in the past 30 days. Approximately half of the participants used ≥5 medications (polypharmacy), with higher prevalence among older patients, women, non-Hispanic whites, those who were overweight, and patients with low socioeconomic status and comorbidities. An increasing trend in polypharmacy prevalence was observed from 36.1% [31.7%-38.7%] in 1999-2002 to 49.6% [47.1%-51.3%] in 2015-2018 (p for trend <0.001). Except for traditional diabetes management drugs, analgesics (20.5% [19.5%-22.3%]), antidepressants (18.0% [17.7%-19.1%]) and proton pump inhibitors (16.0% [15.6%-17.4%]) were commonly used. Among patients with polypharmacy, use of analgesics (33.5% [31.4%-36.6%]), antidepressants (31.5% [29.8%-34.3%]) and proton pump inhibitors (28.3% [26.1%-30.2%]) were common. The proportion of drugs not for hypertension, hyperlipidemia, and diabetes mellitus were substantial (39.7% [38.5%-42.1%]) of the medication burden, with analgesics contributing to 7.6% [6.1%-8.3%] and antidepressants to 5.2% [4.7%-6.3%].
CONCLUSIONS: Significant increases in prescription drug use and polypharmacy were observed among patients with T2D. The prevalence and burden of medications not traditionally monitored by endocrinologists was relatively high, especially in patients with polypharmacy. This finding highlights the polypharmacy issue among these patients and more attentions should be paid to medications not traditionally monitored by endocrinologists.
P54: Patient-Reported & Health Economic Outcomes for Low-Value Medications in Patients Living with Dementia
10:15AM - 10:30AM
Platen M 1 , Rädke A1 , Mohr W1 , Buchholz M1 , Hoffmann W2 , Michalowsky B1 1 German Center for Neurodegenerative Diseases (DZNE), Bonn, NW, Germany, 2 Institute for Community Medicine, University Medicine Greifswald, Greifswald, MV, Germany
OBJECTIVES:
Low-value medications (Lvm) provide little or no benefit to patients, can be harmful, and waste healthcare resources and costs. Although there is evidence that people with dementia (PwD) are particularly affected by Lvm, the long-term impact on their patient-reported and health-economic outcomes has rarely been reported. Therefore, we aimed to analyze the impact of Lvm on health-related quality of life (HRQoL), hospitalizations, and healthcare costs over two years.
METHODS:
This longitudinal analysis was based on baseline, 12 and 24 months follow-up data from 352 community-dwelling PwD. Medications were classified as low-value if they were specifically not recommended in dementia-specific guidelines or published decision aids for clinicians. The 12-item Short-Form Health Survey was used to assess patients' HRQoL. Hospitalizations were assessed retrospectively for 12 months. Direct healthcare costs were monetized using standardized unit costs. Multiple panel-specific regression models were used to analyze the impact on patient-related and health-economic outcomes.
RESULTS:
Over a 2-year period, 52% (n=182) of PwD received Lvm at least once, and 16% (n=56) continuously. PwD, who received Lvm significantly increased the risk of hospitalization by 49% (odds ratio, confidence interval [CI] 95% 1.06–2.09;
p = 0.022), increased health care costs from the payer perspective by €6,810 (CI 95% −707€–14,27€;
p = 0.076), and reduced patients' HRQoL (
b = −1.55; CI 95% −2.76 to −0.35;
p = 0.011) compared to PwD without Lvm during the stated study period.
CONCLUSIONS:
Over one in two community-dwelling PwD received Lvm in 24 months, negatively impacting patient-reported HRQoL, hospitalizations, and direct medical care costs. Appropriate and effective approaches are required to encourage prescribers to avoid Lvm in dementia care wherever possible. Furthermore, adequate alternative treatments are needed as early as possible in the patient journey through the health care system to avoid downstream effects for patients and resource-burdening for health systems.
P53: Description of Polypharmacy and PIM Use Trends in the Elderly From the Perspective of Deprescribing
10:00AM - 10:15AM
Pan S1 , Li S2 , Jiang S3 , Wu H4 , Lyu B 5 1 Guizhou Medical University, Guiyang, Guizhou, China, 2 School of International Pharmaceutical Business, China Pharmaceutical University, Nanjing, Jiangsu, China, 3 Peking University, Beijing, China, 4 Guizhou Medical University, guiyang, guizhou, China, 5 Peking University, Beijing, Beijing, China
OBJECTIVES: The issue of polypharmacy and inappropriate medication use has long been recognized. Interventions such as deprescription have been proposed but the uptake of these interventions is mixed. We aimed to explore changes of overall prescription medication pattern and use of potentially inappropriate medication in the elderly and identify predictors of medication use.
METHODS: We utilized data from participants ≥ 65 years in the National Health and Nutrition Examination Survey (NHANES) between 2011 and 2018. The outcomes included the prevalence of polypharmacy (≥ 5 prescription medications), excessive polypharmacy (≥ 10 prescription medications), medication classes, and potentially inappropriate medications (PIM).
RESULTS: From 2011 to 2018, the prevalence of polypharmacy increased among the elderly (39.2% to 44.2%). The prevalence of excessive polypharmacy remained stable at around 7%. Among participants who were >= 80 years, non-Hispanic black, with lower education, lower income, uninsured, with obesity and comorbidities were more likely to have polypharmacy. The most commonly used medications remained to be antihypertensive (90%), statins (68%), and central nervous system medication (52%). The three classes of medications were the main contributors of medication burden among patients with polypharmacy. The prevalence of PIM increased from 38.9% in 2011-2012 to 40.5% in 2017-2018. Female and participants with lower education, lower income, obesity, uninsured, and comorbidities are more likely to have PIM. Potentially inappropriate use of gastrointestinal medications were the mian PIM.
CONCLUSIONS: The issue of polypharmacy and inappropriate medications use among the elderly remained common and even increased in recent era, especially among population >=80 years old, disadvantaged, and with comorbidities. Our results showed that the uptake of interventions such as deprescription may be limited and called for better strategies to reduce polypharmacy and PIM.
P55: Prevalence and Predictors of Primary Non-Adherence to Medications Prescribed in Primary Care
10:30AM - 10:45AM
Zeitouny S
OBJECTIVES: To study primary nonadherence to medications prescribed in primary care, defined as failure to dispense a new medication or its equivalent within six months of the prescription issue date.
METHODS: We applied a novel data linkage between prescribing data from primary care electronic medical records with comprehensive pharmacy dispensing claims between January 2013 and April 2019 in British Columbia (BC) to estimate primary medication nonadherence and its predictors. We used hierarchical multivariable logistic regression to determine prescriber, patient and medication factors associated with primary medication nonadherence among community-dwelling patients in primary care.
RESULTS: Among 150,565 new prescriptions to 34,243 patients, 17% of prescriptions were never filled. Primary medication nonadherence was highest for drugs prescribed mostly on an as-needed basis, including topical corticosteroids (35.1%) and antihistamines (23.4%). In multivariable analysis, primary medication nonadherence was lower for prescriptions issued by male prescribers (OR 0.66, 95% CI 0.50 to 0.88). Primary medication nonadherence decreased with patient age (OR 0.91, 95% CI 0.90 to 0.92 for each additional 10 years), but increased with polypharmacy among patients 65 or older. Patients filled over 82% of their medication prescriptions within two weeks after their primary care provider visit.
CONCLUSIONS: Prevalence of primary medication nonadherence to new prescriptions was 17%. Interventions to address primary medication nonadherence may want to target older patients with multiple medication use and within the first two weeks of the prescription issue date.
Challenges in Modeling Oncology Outcomes
Digital Conference Pass
Correctly modeling the outcomes of new or existing oncological drugs is of importance for decision making at several levels. However, since data are quite often immature and several accompanying lines of therapy may have followed after initial treatment, a couple of challenges in approaching extrapolations or lines of therapy exist. In this session, presenters will give an overview of several frequently experienced issues and novel ways to approach these.
Moderator
Sofie Berghuis, BSc, MSc, PhD
GSK, Utrecht, Netherlands
P51: Testing a Survival Extrapolation Algorithm for Cancer Immunotherapies: Pass or Fail?
10:30AM - 10:45AM
Latimer N 1 , Taylor K2 , Hatswell A3 , Ho S4 , Okorogheye G4 , Chen C4 , Kim I5 , Borrill J4 , Bertwistle D4 1 University of Sheffield & Delta Hat Limited, Sheffield, DBY, Great Britain, 2 Delta Hat Limited, Long Eaton, NGM, UK, 3 Delta Hat Ltd, Nottingham, UK, 4 Bristol Myers Squibb, Uxbridge, UK, 5 Bristol Myers Squibb, Lawrenceville, NJ, USA
OBJECTIVES: Accurately extrapolating survival beyond trial follow-up is essential in health technology assessment, with model choice often substantially impacting estimates of clinical benefits and cost-effectiveness. Immuno-oncology is especially affected because survival curves can flatten over time, suggesting durable long-term benefits. Recently, Palmer et al. (2022) developed an algorithm to aid immunotherapy survival model selection. We present a practical demonstration of this algorithm using multiple data-cuts from the CheckMate-649 (CM-649) study. We aimed to assess the practical applicability of the algorithm, and whether it identified survival models fitted to earlier data-cuts, that accurately predicted outcomes observed in later data-cuts.
METHODS: The Palmer et al. algorithm was used to: (i) identify candidate survival models given external data, previously expressed expert beliefs, and diagnostic analyses undertaken on the CM-649 data, and (ii) to define plausibility criteria which models must satisfy to be considered credible. Candidate models were applied to 12- and 24-month data-cuts, and predictions compared to plausibility criteria and outcomes observed in longer-term follow-up.
RESULTS: The algorithm was simple to use and offered a systematic procedure for model selection, encouraging highly detailed analyses and ensuring that crucial stages in the selection process were not overlooked. In our case study, log normal, log-logistic, Generalized Gamma, cubic spline, and cure models were identified as candidate models. Of these models, only log-logistic and non-mixture cure models (with cure assumed at 10-15 years post baseline) provided survival estimates that met plausibility criteria. Log-logistic models appeared to under-estimate survival observed in the 36-month data-cut, whereas non-mixture cure models performed well.
CONCLUSIONS: The Palmer et al. algorithm provides a systematic framework for identifying suitable survival models, and for defining plausibility criteria for extrapolation validity. The algorithm requires that model selection is based on explicit justification and evidence. Use of this approach could reduce discordance in technology appraisals.
P49: Comparability of Overall Survival in Real-World and Clinical Trial Data for BRAF+ Advanced Melanoma
10:00AM - 10:15AM
Sajeev G1 , Chen K2 , In G3 , Simpson R4 , Kalia S4 , Christensen D5 , Liu D4 , Rezai N6 , di Pietro A7 , Signorovitch J 4 1 Analysis Group, Inc., Norwood, MA, USA, 2 Pfizer Inc., Cambridge, MA, USA, 3 University of Southern California, Norris Comprehensive Cancer Center, Los Angeles, CA, USA, 4 Analysis Group, Inc., Boston, MA, USA, 5 Analysis Group, Inc., New York, NY, USA, 6 Pfizer Inc., New York, NY, USA, 7 Pfizer SRL, Milan, Italy
OBJECTIVES: External controls based on real-world data (RWD) are increasingly used to complement clinical trial data when assessing effectiveness of treatments in oncology. In the absence of randomization, comparisons between clinical trial and RWD have well-recognized risk of bias, arising from potential differences across these settings in patient populations, diagnostic testing, background therapies and outcome assessments. Suitability of RWD as external controls should be determined on a case-by-case basis considering the RWD source, study outcomes compared, and study design and analysis steps employed to address bias. Empirical assessment of consistency in outcomes across trial and RWD settings can also help assess suitability of external controls based on RWD.
METHODS: We compared overall survival (OS) in patients with metastatic BRAF V600-mutant melanoma treated with encorafenib plus binimetinib (ENCO+BINI) drawn from the phase 3 COLUMBUS trial and from Flatiron Health, an electronic medical records-derived database of primarily community oncology clinics in the US.
RESULTS: After applying key trial inclusion/exclusion criteria to the RWD, imputing missing baseline prognostic factors using multiple imputation, and adjusting for baseline prognostic factors, OS was similar in ENCO+BINI-initiating patients across trial (n=192) and RWD (n=83; hazard ratio (HR): 1.03; 95% CI: 0.53, 1.54) settings. Similar OS between trials (n=241) and RWD (n=816) from Flatiron was also reported in a previous analysis of patients with metastatic melanoma, regardless of molecular testing status, treated with ipilimumab (HR: 0.98 (0.75, 1.26)).
CONCLUSIONS: These findings of consistent OS with clinical trials after applying equivalent I/E criteria, and adjusting for differences in patient profiles and missing data are encouraging for the use of RWD from Flatiron to construct external controls for OS in BRAF+ metastatic melanoma. RWD may augment randomized control arms, provide a reference arm in long-term extension periods, contextualize trial outcomes, or be pooled with trial data to facilitate analyses based on more comprehensive evidence.
P52: Validation of an Algorithm to Identify Lines of Therapy Among Adult Patients With Endometrial and Ovarian Cancer in a Real-World Dataset
10:45AM - 11:00AM
Luhar S 1 , Wallis J2 , Picariello F3 , Tunaru F4 , Hatton G3 , Carpenter L3 1 Arcturis Data, London, UK, 2 Arcturis Data, Kidlington, UK, 3 Arcturis Data, Oxford, UK, 4 Arcturis Data, Orpington, LON, UK
OBJECTIVES: As electronic health records (EHR) do not explicitly capture lines of therapy ( LoT ) received by patients, algorithms a im to automatically extract this information. However, LoT algorithms depend on several arbitrary assumptions. We aim to validate a LoT algorithm in patients with a gynaecological cancer diagnosis using EHR .
METHODS: This validation study use s de- identified EHR for Ovarian and Endometrial cancer patients diagnosed between 200 7 and 20 23 from UK NHS partners collated as part of the Arcturis Data Platform. Upon random allocation of patients to either a baseline or refinement cohor t , we will test variations of the algorithm’s parameters . Appraisal of various parameter combination s will be expressed via the percentage agreement of the number of lines and r egimen definition between the output and a gold standard of blinded manual review . Anticipated a reas of disagreement between manual review and the algorithm include the 120-day gap in treatment administrations that would advance a line ; the 30-day window group ing agents that would indicate a combination therapy line ; and differences in the regimen definition, especially if a drug is d iscontinued shortly after the commencement o f a line.
RESULTS: From a total of 4,757 newly diagnosed o varian cancer patients, and 3,884 newly diagnosed cancer patients , 1582 and 627 , respectively, received chemotherapy. Preliminary analysis indicates that changes to the 120 -day gap in therapy to between 90 and 180 days alters the percentage receiving only one therapy line by <2% ; reducing the gap to 30 days minimally increases the percentage receiving 5+ lines by 4%. Likewise, algorithm o utput is robust to variations in the 30-day line regimen window with negligible difference in output between 30- and 90-day s .
CONCLUSIONS: Initial results indicate minimal sensitivity of the algorithm to reasonable variations in the gap in therapy and line regimen window . Further validation as per the methodology is needed.
P50: Joint Modelling of Intermediate Longitudinal Biomarkers to Predict Overall Survival in Patients with Solid Tumors
10:15AM - 10:30AM
Linsell L 1 , Paracha N2 , Grossman J3 , Bokemeyer C4 , Garcia-Foncillas J5 , Italiano A6 , Vassal G7 , Chen Y1 , Torlinska B8 , Abrams K9 1 Visible Analytics, Oxford, OXF, UK, 2 Bayer Pharmaceuticals, Basel, Switzerland, 3 Bayer Pharmaceuticals, Westerville, OH, USA, 4 University Medical Centre Hamburg, Eppendorf, Hamburg, Germany, 5 University Cancer Institute and the Department of Oncology, Madrid, Spain, 6 Institut Bergonié Comprehensive Cancer Centre, Bordeaux, France, 7 Gustave Roussy Comprehensive Cancer Centre, Villejuif, France, 8 Visible Analytics, Oxford, Oxfordshire, UK, 9 Warwick University, Coventry, WAR, UK
OBJECTIVES: Joint modelling (JM) of longitudinal and time-to-event data simultaneously can provide an estimated biomarker profile (adjusted for informative dropout due to death) or predictions of the time-to-event outcome conditional upon the longitudinal biomarker profile. This application explores the association between tumor burden and overall survival (OS) in patients with solid tumors, compared to a traditional parametric approach.
METHODS: Data were pooled from three phase I/II open-label trials evaluating the safety and efficacy of Larotrectinib in adults and pediatric patients with TRK fusion cancer (data-cut 20/07/2021; n=196). Tumor burden was measured as the sum of diameters of target lesions (SLD). Bayesian joint modelling was used to obtain patient-specific predictions of OS using individual-level SLD profiles up to the time at which the patient died or was censored, using alternative assumptions for the association parameter. These were compared to predictions from a standard Weibull model. All models were adjusted for age, ECOG status >1, metastatic progression at treatment initiation and tumor type.
RESULTS: Median follow-up was 32.4 months (range 0.4 to 71) and 58/196 deaths (29.6%) were observed. The JM using a common association parameter across primary tumor type was the best fitting model. The restricted mean survival time was 8.9 years (95% credible interval (CrI): 6.3 to 11.5) compared to 8.0 years (95% CrI: 5.0 to 13.3) in the Weibull model. 10-year OS predictions from treatment initiation were also similar, with less uncertainty in the JM; 26.4% (95% Crl: 18.0% to 34.8%) compared to 27.7% (95% Crl: 12.8% to 40.4%) in the Weibull model.
CONCLUSIONS: JM can offer an alternative approach to traditional survival modelling and may improve survival predictions from limited follow-up data. This approach allows complex hierarchical data structures, such as patients nested within tumor types. It can also incorporate multiple longitudinal biomarkers in a multivariate modelling framework.
Case Studies
Rare Disease Case Studies
These case studies include improving patient care for Multiple Sclerosis through proximity-oriented approaches in Italy, utilizing real-world evidence for health technology assessment in sickle cell disease in the UK, using patient registries to inform HTA in rare diseases in Brazil, and addressing challenges in using registries for research and patient outcomes in European Haemophilia Consortium.
Moderator
Mariya Kamusheva, M.Ph, PhD
Medical University of Sofia, Sofia, 22, Bulgaria
Prof. Maria Kamusheva holds a master’s degree in Pharmacy from the Faculty of Pharmacy, Medical University-Sofia in 2011. In 2014, she defended her Ph.D. thesis "Pharmacoeconomics of the Treatment of Rare Diseases". She has 8 years of practical experience in community pharmacy settings and almost 2 years in the field of clinical trials. Currently, assoc. prof. Maria Kamusheva works at the Department of Organization and Economics of Pharmacy, Faculty of Pharmacy, Medical University of Sofia.
She carried out over 70 scientific publications (h-index=11; Web of Science, 2023) and participated in congresses, conferences, international training, national and international projects. Assoc. prof. Kamusheva is a member of ISPOR, Bulgarian Pharmaceutical Union, Bulgarian Pharmaceutical Science Society, and Bulgarian Association of Drug Information. Seven pharmacy students defended their master thesis under her supervision. To the current date, she is supervising four Ph.D. students. Her scientific interests are related to pharmacoeconomic, drug utilization, rare diseases, orphan drugs, and pharmaceutical legislation.
CS22: Ensuring Patient Safety Within ‘Managed Access’ – Crizanlizumab to Prevent Recurrent Sickle Cell Crises as a Worked Example
10:15AM - 10:30AM
Patrick H 1 , Bird C2 1 NICE, Manchester, UK, 2 National Institute for Health and Care Excellence, Manchester, Lancashire, UK
Problem Statement: Promising clinical evidence suggested that crizanlizumab can reduce the incidence of vaso-occlusive crises (VOC) in patients with sickle cell disease. There is uncertainty about its long-term (including after stopping the treatment) and cost effectiveness. The National Institute for Health and Care Excellence (NICE UK), recommended it should be used in the NHS in England only as part of a Managed Access Agreement (MAA). The MAA permits time-limited access to crizanlizumab while additional real world and trial data are collected to address uncertainties.
Description: Sickle cell disease is a blood disorder characterised by sickling of the red cells, clumping and blockages in the small blood vessels (VOCs). Crizanlizumab prevents interactions between endothelial cells and blood components involved in the blockages.
NICE estimated that over 450 in England could benefit from access to the drug in a MAA.
Interim trial data shows no statistically significant difference compared with placebo in annualised rates of sickle cell crises. The European Medicines Agency (EMA) recommended revoking the marketing authorisation for crizanlizumab, finding that the benefits of the medicine ‘did not outweigh its risks’.
NICE’s MA programme requires regular evidence updates and monitoring reports (safety and efficacy). NICE's Patient Safety Oversight Group (PSOG) required interim review of the MAA. In addition to the emerging evidence on efficacy it identified a significant drop out rate with some people experiencing an increase in VOCs (a safety concern).
Lessons Learned: Accelerated access to promising new technologies may offer benefit to patients through ‘managed access’ whilst evidence is developed. Whilst effectiveness is the focus, safety monitoring of technologies early in their evidential pathway is important. Rapid healthcare technology evaluation requires robust mechanisms in place to support identification of safety signals and appropriate response.
Stakeholder perspective: This is being told from the perspective of the Patient Safety Oversight Group of an arms-length non-department government body, NICE.
CS21: Integrating Proximity Care in Multiple Sclerosis Patient Management in Italy: The Stayhome Project Case Study
10:00AM - 10:15AM
Filippi M1 , Gallo P2 , Gasperini C3 , Marfia GA4 , Avolio C5 , Bergamaschi R6 , Capobianco MA7 , Dotta M8 , Grimaldi L9 , Lus G10 , Patti F11 , Pucci E12 , Quatrale R13 , Solla P14 , Bandiera P15 , Panetta C16 , Parretti S17 , Lo Muto R18 , Pinto L 19 1 IRCCS San Raffaele Scientific Institute, Milan, Milan, Italy, 2 Azienda Ospedaliera Universitaria di Padova, Padova, Italy, 3 Azienda Ospedaliera San Camillo Forlanini, Rome, Rome, Italy, 4 Fondazione Policlinico Tor Veragata, Rome, Rome, Italy, 5 Policlinico di Foggia, Foggia, Foggia, Italy, 6 IRCCS Mondino Foundation, Pavia, Italy, 7 Azienda Ospedaliera Santa Croce e Carle, Cuneo, Cuneo, Italy, 8 ASL Alba-Bra, Alba, Alba, Italy, 9 Fondazione Istituto G. Giglio, Cefalù, Italy, 10 Seconda Università degli Studi di Napoli, Napoli, Napoli, Italy, 11 Azienda Ospedaliera Universitaria Policlinico "G. Rodolico - S. Marco", Catania, Catania, Italy, 12 Ospedale Augusto Murri, Fermo, Fermo, Italy, 13 Ospedale dell'Angelo di Mestre, Venezia, Venezia, Italy, 14 Azienda Ospedaliera Universitaria di Sassari, Sassari, Sassari, Italy, 15 Associazione Italiana Sclerosi Multipla, Genova, Genova, Italy, 16 IQVIA Solutions Italy S.r.l., Milan, Milan, Italy, 17 IQVIA Solutions Italy S.r.l., Peschiera Borromeo, MI, Italy, 18 Biogen Italy, Milan, Milan, Italy, 19 IQVIA Solutions Italy S.r.l., Milano, MI, Italy
Problem Statement: In Italy, around 133,000 people live with Multiple Sclerosis (MS). MS patients benefit from an ultra-specialized approach but face organizational complexity due to a lack of proximity-oriented approaches.
Description: StayHome project aims at supporting Centers in the integration of proximity care in the management of MS patients, in line with recent healthcare legislation and investments. First, a mixed method survey has been conducted involving 50 Centers to identify the main improvement areas for proximity care implementation and to build a cross-Center analysis framework. 14 MS Centers in 9 Regions were then involved on a voluntary basis to map the AS IS and define a TO BE care model. Different areas of intervention have been identified (e.g., formalization and digitalization of integrated Hospital – Territory pathways). A Steering Committee, including Key Opinion Leaders, PAG (Patient Association Group) and Scientific Association representatives, supervised progresses and methodologies adopted in the project, including Key Performance Indicators to monitor the patient pathway and a Maturity Model to assess and support MS Centers in the path towards proximity care. More than 150 healthcare professionals, hospital managers and regional stakeholders have been involved with a potential impact for about 14,000 patients.
Lessons Learned: Implementation of proximity care in MS is challenging due to the complexity of the disease. A unique approach to implement proximity care in MS patient pathways results to be suboptimal, due to the impact of regional and local specificities. A bottom-up approach results to be more effective, when designed with healthcare professionals and patient associations, and when hospital and regional managers are onboard. These aspects should be considered before scaling up proximity care models at regional and national level. Anyway, the Maturity Model validated provides building blocks and good practices to support proximity care implementation in MS.
Stakeholder perspective: Provider and PAG
CS24: Creating Alignment Across Registries to Optimize for Real-World Evidence in Rare Bleeding Disorders (RBD): Outcomes From the EHC Think Tank Registries Workstream
10:45AM - 11:00AM
Savini L, Skouw-Rasmussen N
Problem Statement: Registries are key for driving research in rare diseases with large datasets and using real-world evidence to inform patient outcomes. Their use in clinical care and research is established, and they are increasingly used for regulatory and economic assessments of novel therapies. However challenges prevent them to be used to their full potential.
Description: A multi-stakeholder workstream on registries was established by the European Haemophilia Consortium (EHC) Think Tank to discuss these issues and propose solutions. Its members convened over two years, culminating in an innovation summit. We present here the outcomes of this work and identified solutions/trends.
Lessons Learned : Stakeholders identified the following as barriers to registries use: fragmentation of multiple data sources creates challenges associated with consensus on data sets, secure access, interoperability, multiplicity of data, data quality and delivery. Stakeholders agree that no additional RBD registry should be established. The significance of change relies on international cross-border rights- and responsibility-based guidance aligning the variety of national and regional registries. Beyond the technical aspect, safeguarding and making registries fit for the future depends on trust, collaboration and standardised data collection and reporting. The question of sustainable financing for optimal operational use remains. The workstream agrees that participation to data collection via registries is an integral part of patient care.
Stakeholder perspective : The EHC Think Tank Registries Workstream brings together clinicians, regulators, health economists, health policy specialists, patient representatives, the pharmaceutical industry, registry owners and health technology specialists.
CS23: RWD From Patient Registry for Rare Diseases and Its Uses in HTA Process in Brazil
10:30AM - 10:45AM
Biglia LV 1 , Ribeiro TB 2 1 Takeda Brasil, São Paulo, SP, Brazil, 2 Takeda Brasil, São Paulo, Brazil
Problem Statement: The Brazilian Health Technology Assessment (HTA) agency−Conitec, since 2012 provides recommendations for local Universal Healthcare System (SUS). Reimbursement of rare diseases technologies is challenging due to uncertainties in scientific evidence, high treatment cost and lack of specific rare diseases HTA guidance. In this context, real word evidence (RWE) might be an important source to add information to HTA process. We aim to describe an experience of RWE used in recent Brazilian HTA submission for Fabry disease and Hereditary angioedema (HAE).
Description: Fabry Outcome Survey (FOS) and Icatibant Outcome Survey (IOS) are diseases registries sponsored by Takeda that collect information on patients with Fabry (using agalsidase-alfa) and HAE (using Icatibant), respectively.
FOS was initiated in April 2001 and concluded in September 2022 with 4,480 patients enrolled from 109 centers across Brazil and 23 other countries. The registry aimed to follow renal function, cardiovascular and neurological effects, pain and QoL.
IOS was initiated in July 2009, in March 2019, 1,052 patients have been enrolled from Brazil and 12 other countries, and assessed outcomes of attack-related frequency, severity, and clinically significant relief of symptoms for Icatibant.
Agalsidase-alfa and Icatibant were rejected by Conitec in 2020 and 2015, respectively. The new submissions (2022) did not include novel RCT data , thus including FOS and IOS data, the clinical dossier added long-term effectiveness and safety data. Agalsidase-alfa was recently incorporated by Conitec-SUS and Icatibant HTA-process is ongoing.
Lessons Learned:
Stakeholder perspective:
Breakouts: IP, WS, & OBS
The Finose Collaboration - Joint Health Technology Assessment in the Nordics
Digital Conference Pass
Level: Introductory
PURPOSE: To present the FINOSE collaboration formed by four Nordic countries to perform joint health technology assessment (HTA), one of several regional activities created to collaborate on joint assessments. DESCRIPTION: There has long been a growing number of new pharmaceuticals in need of HTA for reimbursement decisions, and there is no sign of the numbers declining in the future. Therefore, HTA agencies are looking for more efficient ways to conduct assessments and make reimbursement decisions while maintaining the rigor and quality of the assessments. One way to achieve this is through joint HTA within Europe or between countries, which allows for sharing of resources and knowledge between HTA agencies. FINOSE is one such initiative, launched in 2018 by the Finnish Medicines Agency (Fimea), the Norwegian Medicines Agency (NoMA), and the Dental and Pharmaceutical Benefits Agency (TLV) in Sweden. In May 2023, the collaboration was expanded by the inclusion of the Danish Medicines Council (DMC). While the European regulation on HTA focuses on the requirements of HTAs with respect to clinical evidence, FINOSE focuses primarily on the health economic assessment. The FINOSE process has now been used for a number of joint HTA reports. The reports have been used for price negotiations and national decision-making in the respective countries. The discussion lead will start by giving a short introduction and introducing the presenters. The presenters will provide an overview of the FINOSE process, learnings from completed projects, as well as the outlook for FINOSE going forward. The session will benefit those who are working on submissions to the Nordic countries or those who would like to identify learnings for joint appraisals in other countries and will finish with a panel discussion and audience participation.
Speakers
Ehm Galijatovic, Phd
Danish Medicines Council (DMC), Solrød Strand, Denmark
I work as a Chief Health Scientific Officer in the Danish Medicines Council. I represent the Danish Medicines Council in FINOSE.
6 years of experience in leading health technology assessments in various disease areas. Strong interest in methodological issues, international collaborations and prioritization in the Danish health care system.
Before working with HTA, I spent approximately 6 years working with epidemiological studies including PhD in genetic epidemiology from University of Copenhagen, Faculty of Health and Medical Sciences.
In my sparetime, you can always find me and my family on the football field, watching our boys play football.
Johan Pontén, MA
Medicine Evaluation Committee (MEDEV), Stockholm, Sweden
Johan Pontén is Senior Manager of International Affairs at The Dental and Pharmaceutical Benefits Agency in Sweden since 2014. He is since 2021 co-chair of the Medical Evaluation Committee, MEDEV, a network of 22 national authorities from 18 Member States and Norway bringing together all the relevant institutions (national HTA agencies and social health insurers-payers) responsible for the assessment, pricing and reimbursement of medicines in Europe. He has also supported the FINOSE collaboration between Nordic HTA agencies from the start in 2017. He has had a career in the Swedish Public Service and holds a Master in Pedagogic Leadership.
Anja Schiel, PhD
Norwegian Medicines Agency (NoMA), Oslo, 02, Norway
Anja Schiel works as Special Adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects at the Norwegian Medicines Agency. She has been Chair of EMA’s Biostatistics Working Party 2017 – 2019 and from 2019 – 2022 she was Chair of the Scientific Advice Working Party (SAWP) at EMA. She continues currently as alternate member at the SAWP and is member of the new Methodology Working Party (MWP) recently established at EMA.
She has been heavily involved in EUnetHTA JA3 and its successor, EUnetHTA 21 with a particular focus on parallel EMA-HTA scientific advices.
Discussion Leaders
Thor-Henrik Brodtkorb, PhD
RTI Health Solutions, Ljungskile, O, Sweden
Thor-Henrik Brodtkorb, PhD, is executive director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 12 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision-analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and ISPOR.
At RTI-HS, Dr. Brodtkorb leads the development of cost-effectiveness, cost-utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a co-author of the book “Budget-Impact Analysis of Health Care Interventions: A Practical Guide.”
Basket Weaving: Can Bayes Help Us Develop a Better Path for Evaluating Efficacy in Basket Trials?
Digital Conference Pass
Level: Intermediate
PURPOSE: As drug development efforts in oncology focus on targeting increasingly rare driver mutations, new challenges for evaluating treatment efficacy are introduced. The rarity of some driver mutations presents difficulty in recruiting sufficient numbers of clinical trial patients without widening eligibility. Basket trials are increasingly being used to evaluate treatment responses for patients with a variety of different tumour histologies that share a common targetable mutation. Evaluation of outcomes for these “histology-independent therapies” in basket trial settings presents a challenge as outcomes might be heterogeneous across histologies. As these studies are usually single-arm trials, evaluating the comparative effectiveness of the treatment under study requires indirect comparisons. An overview of the challenges of evaluating both non-comparative and comparative efficacy in basket trials will be provided. Discussants will outline how Bayesian hierarchical modelling approaches have been applied to address these challenges and how they may play a role in HTA decision-making.
DESCRIPTION: Bart Heeg will introduce the topic and speakers (10 minutes). Samantha Wilkinson will provide some background on the rationale and growing use of basket trials in oncology, and outline challenges faced by pharmaceutical companies (15 minutes). Sofia Dias will illustrate some of the challenges with evaluating efficacy in basket trials, discussing (i) the pros and cons of analysis approaches which ignore cross-histology heterogeneity by pooling together the data for multiple histologies versus no-pooling approaches, and (ii) the difficulty of performing unbiased indirect treatment comparisons (15 minutes). Emma Mackay will discuss how Bayesian hierarchical modelling approaches can improve upon complete pooling and no-pooling approaches, and can be used to mitigate confounding in indirect treatment comparisons while preserving limited precision/power due to small sample sizes (15 minutes). We will utilize a live smartphone-based polling feature during the workshop to foster active participation and make the learning process more engaging.
Discussion Leaders
Bart Heeg, MSc, PhD
Cytel Inc., Rotterdam, ZH, Netherlands
Bart is Vice President HEOR at CYTEL. Bart has a PhD in health economics. He has over 20 years of experience in heath economic and outcomes consulting. He focusses on indirect treatment comparisons, surrogacy analyses and health economic modelling.
Discussants
Sofia Dias, PhD
University of York, York, YOR, United Kingdom
Sofia is Professor of Health Technology Assessment at the University of York (UK) and Director of the CRD/CHE Technology Assessment Group that delivers technology assessment reports for the National Institute for Health and Care Excellence (NICE) through York's NIHR funded TAR programme. Sofia is also a member of NICE Technology Appraisals Committee D.
She is a statistician with interests in Bayesian methods for evidence synthesis and their application to decision-making. Of particular interest are methods for network meta-analysis, bias-adjustment, synthesis of related outcomes and population-adjusted treatment comparisons.
Sofia has collaborated with the NICE Decision Support Unit to produce several technical support documents which provide guidance to all those involved in submitting or critiquing evidence as part of NICE Technology Appraisals and is lead author of the Wiley book on Network Meta-analysis for Decision Making. She also has a wide experience of developing and applying evidence synthesis methods in NICE clinical guidelines and Cochrane Reviews.
Emma Mackay, MA, MSc
Cytel, Toronto, ON, Canada
Emma Mackay is a Research Principal (Statistics) in the Real World and Advanced Analytics group at Cytel, specializing in Bayesian methods. Her recent work has focused on the application of Bayesian borrowing methods to challenges of evaluating efficacy/effectiveness in rare disease settings. She has extensive experience with causal inference methods for both clinical trials and real world data, and in latent variable methods, survival modelling and meta-analysis.
10:00 - 11:15
Spotlight Session
Behavioral Economics to Inform Healthcare Decisions
Digital Conference Pass
Behavioral economics can significantly inform healthcare decisions by recognizing and addressing the cognitive biases and psychological factors that influence patient behavior and healthcare choices. By framing health information in a way that emphasizes potential losses or providing immediate rewards for preventive actions can encourage individuals to adopt healthier lifestyles and adhere to treatment plans. Moreover, behavioral economics can help optimize healthcare systems by improving patient engagement, enhancing decision-making processes, and ultimately leading to better health outcomes and resource allocation. This session will include an overview of behavioral economics and examples of how it’s applications can inform treatment decisions and improve outcomes within chronic diseases.
Additional speakers to be added as confirmed.
Moderators
Shelby Reed, PhD, RPh
Duke University’s School of Medicine, Durham, NC, USA
Shelby D. Reed, PhD, is Professor in the Departments of Population Health Sciences and Medicine at Duke University’s School of Medicine. She is the director of the Center for Informing Health Decisions at the Duke Clinical Research Institute. She also is core faculty at the Duke-Margolis Center for Health Policy and an affiliate member at the Duke Cancer Institute. Shelby has 25 years of experience in health economics, health preferences and health policy research, with more than 200 published manuscripts. Shelby has been an active member of ISPOR for over 20 years. She was the first recipient of ISPOR's Bernie O’Brien New Investigator Award in 2005. She was elected to ISPOR’s Board of Directors in 2009. She served as President of ISPOR in 2017-2018 when she helped to establish ISPOR’s Women in HEOR Initiative. Most recently, she served as Chair of the Health Sciences Policy Council from 2019 to 2021.
Speakers
David Asch, MD
University of Pennsylvania, Philadelphia, PA, USA
David A. Asch, MD, MBA is the Sr Vice Dean for Strategic Initiatives at the Perelman School of Medicine of the University of Pennsylvania and the John Morgan Professor at the Perelman School and the Wharton School. He created and from 2001 to 2012 directed the Center for Health Equity Research and Promotion -- the Department of Veterans Affairs national center to examine and eliminate racial disparities in health and health care. From 1998 to 2012 he was Executive Director of the Leonard Davis Institute of Health Economics. From 2012 to 2022 he was Executive Director of the Penn Medicine Center for Health Care Innovation. His research is in the area of behavioral economics and aims to improve how physicians and patients make medical choices.
He has been recognized for contributions to teaching and research, including the VA Under Secretary’s Award for Outstanding Achievement in Health Services Research (2008), the Robert J. Glaser Distinguished Teacher Award from the Association of American Medical Colleges (2009), the John M. Eisenberg Award for Career Achievement in Research from the Society of General Internal Medicine (2010), the David E. Rogers Award from the Association of American Medical Colleges for “major contributions to improving the health and health care of the American people” (2018), the Distinguished Investigator Award (2019) and Distinguished Educator Award (2022) from the Association for Clinical and Translational Science, and the Distinguished Career Award from AcademyHealth (2020). He is an elected member of the Association of American Physicians and the National Academy of Medicine.
Isabelle Huys, PharmD, PhD
Department of Clinical Pharmacology and Pharmacotherapy, KU Leuven, Leuven, VBR, Belgium
Prof. dr. Isabelle Huys is full professor at the University of Leuven (Belgium). She leads the research group on Regulatory Sciences at the Faculty of Pharmaceutical Sciences, embedding the Patient Evidence and Preference Center (PPRC). Her research focusses on market access of medicines and therapies, with particular attention to the inclusion of the voice of the patient in drug development, evaluation, market access and usage. In addition, ethical, legal and social issues (ELSI) are studied in an interdisciplinary context.
Yannick Vandenplas, PhD
HICT, Gent, Belgium
Yannick Vandenplas, PhD graduated as a pharmacist in 2019, after which he obtained his PhD degree in pharmaceutical sciences at the University of Leuven (Belgium).
During his PhD research, he conducted a four-year research project focusing on pharmaceutical policy. In particular, he looked at ways to make off-patent biologicals markets more competitive and sustainable. This project was in close collaboration and funded by the Belgian national health insurer, RIZIV-INAMI. One of his research topics was the application of insights from behavioral economics into pharmaceutical policymaking. He found that there are multiple avenues to integrate behavioral economics’ insights into pharmaceutical policy, especially for policy strategies related to off-patent biologicals and biosimilars.
Today, Yannick works as a health economics consultant at Hict, which is a Belgian consultancy company specialized in market access of pharmaceuticals, including health economics and outcomes research.
11:30 - 12:45
Plenary Session
The Calm Before the Storm? Delivering the New Reality for EU HTA
Digital Conference Pass
EU collaboration has been in the planning by HTA agencies for over 16 years. Since the adoption of the EU HTA Regulation in January 2022, it will become the new way of working for all stakeholders from January 2025. Whilst the Regulation covers many facets, most of the activity, and arguably the most publicly visible component, will be the Joint Clinical Assessments (JCAs). In little over 12 months from now, the first technologies mandated by the new Regulation will be starting their pathways through a JCA. Health Technology Developers & other Stakeholders are already planning for the initial assessments.
The plenary will discuss what the stakeholders require of each other, and together how they can address the anticipated challenges to ensure a smooth adoption of the new Regulation. The discussion will identify the benefits and risks of the new approach, demonstrating what will be required to turn the rhetoric into action. Will the regulation simply result in a re-engineering of the flow of information from developers to decision makers, or is it an opportunity for Europe to have a strategic input to the development of innovative technologies. The plenary will consider what will be required to ensure the new regulation, and specifically the JCAs, deliver the promised efficiencies resulting in better access for patients.
We will discuss:
From The European Commission – What contribution the Commission require from stakeholders to develop the Implementing Acts, to ensure the Regulation is operational & fit for purposeFrom an EU HTA perspective : what the EU Coordination Group require from industry and Member States to make JCA implementation a successFrom an innovator perspective : what technology developers require to be able to re-engineer their internal processes to meet the requirements of the new Regulation and transformed National processesFrom a Member State perspective : what they require from Industry and expect from the EU process to ensure efficiency & predictability in their local processesFor the Patient : What are the opportunities for patients from the new Regulation? What happens to the patient voice in member state processes if given a voice ‘in Europe’?
Moderators
Adrian Griffin, MSc
Johnson & Johnson, Buckinghamshire, United Kingdom
Adrian has worked in the life sciences industry for over 25 years, predominantly in the fields of health economics, outcomes research, and reimbursement policy, with experience across the pharmaceutical, medical device, and diagnostic sectors. His present role is Vice President, HTA and Access Policy at Johnson & Johnson. Adrian graduated in Medicinal Chemistry from University College London, obtained a post-graduate teaching qualification from Oxford University, and a MSc in Health Economics at City University, London.
Speakers
Roisin Adams, MPharm, MSc., PhD
National Centre for Pharmacoeconomics, Dublin, Ireland & Discipline of Pharmacology and Therapeutics, Trinity College Dublin, Dublin, Ireland
Greg Rossi, PhD
AstraZeneca, Cambridge, United Kingdom
Greg Rossi, Ph.D., is the Senior Vice President responsible for the Oncology Business Unit in Europe and Canada at AstraZeneca. He received his doctorate in Biochemical Engineering from University College London (UCL) and has worked since then in the biotechnology/pharmaceutical industry in the clinical development and commercialisation of oncology therapeutics.
Over the last 25 years, Greg has worked in a number of national (US and UK), regional (Europe) and global roles for Amgen, Genentech, Roche and finally AstraZeneca. Before taking over the management of the Europe and Canada oncology business at AstraZeneca, Greg was responsible for leading the global DNA damage response (DDR) portfolio as well as the global Immuno-Oncology franchise.
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
Dr. Ryll holds a medical degree from the Free University of Berlin, Germany, and a PhD in Biomedical Sciences from University College London, UK. After losing her husband to Melanoma, she founded the Melanoma Patient Network Europe and developed a special interest in patient-centric clinical research and innovation, innovative trial designs and novel drug development concepts. Between 2019 and 2021, she was member of the first EU Cancer Mission Board where her particular interest has been how to best leverage the potential of personalized medicine for patients and society through novel forms of collaboration and the support of health policy and governance. Currently, she works as strategist at SIR, Stockholm School of Economics' Research Institute for Mission Zero Cancer, an innovation ecosystem in health financed by Vinnova, Sweden's innovation agency.
Jose Valverde, MD
European Commission, Brussels, Belgium
Dr. Valverde graduated from the University of Seville in 1991 with a degree in medicine and surgery. He specialised in Emergency and Casualty Medicine (1993) and Family and Community Medicine (2012).
He works at the European Commission, DG-CNECT, Unit H.3 eHealth, Wellbeing and Ageing as Policy & Programme Officer, based in Luxembourg. Currently Team Leader of the “1 Million Genomes” Initiative, PO for 8 H2020 funded research projects and Topic Coordinator for Call SC1-DTH-11-2019. Dr. Valverde is also an active member of the European Advisory Committee on Health Research (EACHR) of WHO-Europe.
Previous positions:
Director of the Andalusian Agency for Health Technology Assessment (2000-2004);
International & Research Advisor at the Cabinet of the Andalusia Regional Minister for Health (2004-2006);
Scientific Officer at the European Commission Joint Research Centre (2006-2009);
Director General of the Spanish National Centre for Accessibility Technologies (2010-2011); and
Andalusian Regional Health Department as senior emergency & casualty physician, coordinator, regulator and researcher (2011-2018, 1995-2000).
Dr Valverde´s research has focused on eHealth including Personal Health Systems, Health 2.0, mHealth, and European healthcare systems enabled through Information Society Technologies. Another related research line is on accessibility technologies for active and healthy ageing.
